task_name,split_name,entity_table,entity_id,evidence_domain,evidence_grade,canonical_name,modality,target_gene_symbol,disease_context,category,evidence_label,source_title,leakage_group,split_strategy,version,source_pmid,source_doi
offtarget_safety_v0_1,test,offtarget_evidence,10,offtarget,A,GSK2910546A ASO gapmer panel from PMID 26338776,ASO,BACH1,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization.,source:26338776|molecule:GSK2910546A ASO gapmer panel from PMID 26338776,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,26338776,10.1093/nar/gkv857
offtarget_safety_v0_1,test,offtarget_evidence,60,offtarget,A,siRNA-based,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Targeting CIDEB alleviates liver steatosis and fibrosis in mouse MASH models.,source:9324|molecule:434,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40503175,10.1016/j.omtn.2025.102567
offtarget_safety_v0_1,test,offtarget_evidence,115,offtarget,A,siRNAs,siRNA,,,off-target evidence,off-target evidence,Respiratory Delivery of Highly Conserved Antiviral siRNAs Suppress SARS-CoV-2 Infection.,source:1788|molecule:435,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41373822,10.3390/ijms262311675
offtarget_safety_v0_1,test,offtarget_evidence,135,offtarget,A,siNTC,siRNA,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Small Interfering RNA Therapy Targeting the Long Noncoding RNA SMILR for Therapeutic Intervention in Coronary Artery Bypass Graft Failure.,source:8415|molecule:468,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40928446,10.1016/j.jacbts.2025.101364
offtarget_safety_v0_1,test,offtarget_evidence,222,offtarget,A,"FM10 (recovered B2 from PMID:27378237, curator:CM)",PMO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics.,source:15206|molecule:1004,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,27378237,10.1038/mt.2016.111
offtarget_safety_v0_1,test,offtarget_evidence,344,offtarget,B,VIR-2218,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization.,source:1411|molecule:789,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,35736224,10.1093/nar/gkac539
offtarget_safety_v0_1,test,offtarget_evidence,346,offtarget,B,LNA-PS ASO,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization.,source:2813|molecule:791,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,26338776,10.1093/nar/gkv857
offtarget_safety_v0_1,test,offtarget_evidence,348,offtarget,B,siRNA-604,siRNA,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Profiling of mismatch discrimination in RNAi enabled rational design of allele-specific siRNAs.,source:15837|molecule:793,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,19815667,10.1093/nar/gkp835
offtarget_safety_v0_1,test,offtarget_evidence,451,offtarget,B,IGF2BP2,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,An exosome-biomimetic photothermal nanocarrier for IGF2BP2 siRNA delivery and enhanced ferroptosis in head and neck squamous cell carcinoma.,source:1732|molecule:882,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,41859644,10.1016/j.mtbio.2026.102999
offtarget_safety_v0_1,test,offtarget_evidence,483,offtarget,B,SMILR siRNA,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Small Interfering RNA Therapy Targeting the Long Noncoding RNA SMILR for Therapeutic Intervention in Coronary Artery Bypass Graft Failure.,source:8415|molecule:912,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,40928446,10.1016/j.jacbts.2025.101364
offtarget_safety_v0_1,train,offtarget_evidence,1,offtarget,A,SNA-modified GalNAc-siRNA panel from PMID 41994868,siRNA,TTR,,seed-mediated off-target effect,seed-mediated off-target effect,Acyclic serinol nucleic acid modification of siRNAs overcomes seed region mediated off-target effects while maintaining potency.,source:41994868|molecule:SNA-modified GalNAc-siRNA panel from PMID 41994868,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41994868,10.1093/nar/gkag345
offtarget_safety_v0_1,train,offtarget_evidence,2,offtarget,A,siVIM-270 chemical modification panel from PMID 41467115,siRNA,VIM,,seed-mediated off-target effect,seed-mediated off-target effect,An siRMSD parameter of structural distortion induced by chemical modification is predictive of the off-target effect of siRNA.,source:41467115|molecule:siVIM-270 chemical modification panel from PMID 41467115,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41467115,10.1016/j.omtn.2025.102693
offtarget_safety_v0_1,train,offtarget_evidence,3,offtarget,B,RGLS4326,ASO,,,off-target evidence,off-target evidence,The nucleobase guanine at the 3'-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity.,source:41315228|molecule:RGLS4326,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41315228,10.1038/s41467-025-65799-5
offtarget_safety_v0_1,train,offtarget_evidence,4,offtarget,A,APP-AON/ATXN3-AON/HTT-AON panel from PMID 41066632,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Determining off-target effects of splice-switching antisense oligonucleotides using short read RNAseq in neuronally differentiated human induced pluripotent stem cells.,source:41066632|molecule:APP-AON/ATXN3-AON/HTT-AON panel from PMID 41066632,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41066632,10.1093/hmg/ddaf153
offtarget_safety_v0_1,train,offtarget_evidence,5,offtarget,A,PNA-braced ASO panel from PMID 38042877,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Dynamic and static control of the off-target interactions of antisense oligonucleotides using toehold chemistry.,source:38042877|molecule:PNA-braced ASO panel from PMID 38042877,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,38042877,10.1038/s41467-023-43714-0
offtarget_safety_v0_1,train,offtarget_evidence,6,offtarget,A,DM1 ASO panel from PMID 37200862,ASO,DMPK,,transcriptome-level off-target effect,transcriptome-level off-target effect,Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1.,source:37200862|molecule:DM1 ASO panel from PMID 37200862,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37200862,10.1016/j.omtn.2023.04.010
offtarget_safety_v0_1,train,offtarget_evidence,7,offtarget,A,ALN-HBV/ALN-HBV02 GalNAc-siRNA panel from PMID 35736224,siRNA,HBV,,seed-mediated off-target effect,seed-mediated off-target effect,From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization.,source:35736224|molecule:ALN-HBV/ALN-HBV02 GalNAc-siRNA panel from PMID 35736224,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,35736224,10.1093/nar/gkac539
offtarget_safety_v0_1,train,offtarget_evidence,8,offtarget,A,GalNAc-siRNA panel from PMID 29459660,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity.,source:29459660|molecule:GalNAc-siRNA panel from PMID 29459660,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,29459660,10.1038/s41467-018-02989-4
offtarget_safety_v0_1,train,offtarget_evidence,12,offtarget,B,siRNA candidate from PMID 41020503,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Modified unlocked nucleic acid (MUNA) mitigates off-target effects of small interfering RNAs.,source:41020503|molecule:siRNA candidate from PMID 41020503,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41020503,10.1093/nar/gkaf937
offtarget_safety_v0_1,train,offtarget_evidence,13,offtarget,B,ASO candidate from PMID 36229039,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,INRI-seq enables global cell-free analysis of translation initiation and off-target effects of antisense inhibitors.,source:36229039|molecule:ASO candidate from PMID 36229039,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,36229039,10.1093/nar/gkac838
offtarget_safety_v0_1,train,offtarget_evidence,15,offtarget,B,siRNA candidate from PMID 29246303,siRNA,,,off-target evidence,off-target evidence,Site-Specific Modification Using the 2'-Methoxyethyl Group Improves the Specificity and Activity of siRNAs.,source:29246303|molecule:siRNA candidate from PMID 29246303,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,29246303,10.1016/j.omtn.2017.10.003
offtarget_safety_v0_1,train,offtarget_evidence,16,offtarget,B,siRNA candidate from PMID 41897327,siRNA,,,sequence-specificity/off-target evidence,sequence-specificity/off-target evidence,Selective Silencing of TDP-43 P. G376D Mutation Reverses Key Amyotrophic Lateral Sclerosis-Related Cellular Deficits.,source:41897327|molecule:siRNA candidate from PMID 41897327,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41897327,10.3390/biom16030393
offtarget_safety_v0_1,train,offtarget_evidence,59,offtarget,A,"ASO 6 (recovered B2 from PMID:40672232, curator:CM)",ASO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Targeted RNA editing by direct delivery of an adenosine deaminase-antisense oligo conjugate.,source:1824|molecule:989,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40672232,10.1101/2025.07.11.664364
offtarget_safety_v0_1,train,offtarget_evidence,62,offtarget,A,siRNAs,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Overcoming GNA/RNA base-pairing limitations using isonucleotides improves the pharmacodynamic activity of ESC+ GalNAc-siRNAs.,source:2309|molecule:435,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,34648028,10.1093/nar/gkab916
offtarget_safety_v0_1,train,offtarget_evidence,63,offtarget,A,siRNAs,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,AGO-accessible anticancer siRNAs designed with synergistic miRNA-like activity.,source:12558|molecule:435,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,33664996,10.1016/j.omtn.2021.01.018
offtarget_safety_v0_1,train,offtarget_evidence,64,offtarget,A,unspecified siRNA (PMID:31375812),siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.,source:4213|molecule:990,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,31375812,10.1038/s41587-019-0205-0
offtarget_safety_v0_1,train,offtarget_evidence,65,offtarget,A,unspecified ASO (PMID:29390093),ASO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Acute hepatotoxicity of 2' fluoro-modified 5-10-5 gapmer phosphorothioate oligonucleotides in mice correlates with intracellular protein binding and the loss of DBHS proteins.,source:2060|molecule:991,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,29390093,10.1093/nar/gky060
offtarget_safety_v0_1,train,offtarget_evidence,66,offtarget,A,"Sequence 1 (recovered B2 from PMID:28918038, curator:CM)",ASO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Strategies for In Vivo Screening and Mitigation of Hepatotoxicity Associated with Antisense Drugs.,source:2064|molecule:992,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,28918038,10.1016/j.omtn.2017.07.003
offtarget_safety_v0_1,train,offtarget_evidence,67,offtarget,A,LNA ASO,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts.,source:2062|molecule:438,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,26553810,10.1093/nar/gkv1210
offtarget_safety_v0_1,train,offtarget_evidence,78,offtarget,A,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Integrating transcriptomics and proteomics to assess antisense oligonucleotide safety and efficacy: a time-resolved approach.,source:1035|molecule:986,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41918821,10.1093/narmme/ugag016
offtarget_safety_v0_1,train,offtarget_evidence,91,offtarget,A,"S9 (recovered B2 from PMID:41552385, curator:CM)",siRNA,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment.,source:3301|molecule:994,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41552385,10.1016/j.omtn.2025.102802
offtarget_safety_v0_1,train,offtarget_evidence,113,offtarget,A,unspecified siRNA (PMID:40385635),siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,"Design, pharmacology, and toxicology of a novel chemically modified siRNA targeting hepatic angiotensinogen.",source:1477|molecule:995,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40385635,10.1016/j.omtn.2025.102542
offtarget_safety_v0_1,train,offtarget_evidence,116,offtarget,A,unspecified siRNA (PMID:41341748),siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,A novel bispecific siRNA concept: Efficient dual knockdown of YAP1 and WWTR1 with a single guide strand.,source:1789|molecule:996,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41341748,10.1016/j.omtn.2025.102768
offtarget_safety_v0_1,train,offtarget_evidence,144,offtarget,B,unspecified siRNA (PMID:40142497),miRNA agomir,,,transcriptome-level off-target effect,transcriptome-level off-target effect,In Vitro Transcribed Artificial Primary MicroRNA for the Inhibition of Hepatitis B Virus Gene Expression in Cultured Cells.,source:9881|molecule:1064,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40142497,10.3390/microorganisms13030604
offtarget_safety_v0_1,train,offtarget_evidence,188,offtarget,A,"Glu-CT102MOE5 (recovered B2 from PMID:37251692, curator:CM)",ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Modified ASO conjugates encapsulated with cytidinyl/cationic lipids exhibit more potent and longer-lasting anti-HCC effects.,source:14112|molecule:999,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,37251692,10.1016/j.omtn.2023.04.028
offtarget_safety_v0_1,train,offtarget_evidence,190,offtarget,A,"si-S1A3-Chol (recovered B2 from PMID:36618268, curator:CM)",siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Chemically modified small interfering RNA targeting Hedgehog signaling pathway for rheumatoid arthritis therapy.,source:14301|molecule:1000,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,36618268,10.1016/j.omtn.2022.12.008
offtarget_safety_v0_1,train,offtarget_evidence,193,offtarget,A,unspecified ASO (PMID:36511872),ASO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects.,source:2253|molecule:1001,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,36511872,10.1093/nar/gkac1108
offtarget_safety_v0_1,train,offtarget_evidence,207,offtarget,A,"T39 ASO (recovered B2 from PMID:32180554, curator:CM)",ASO,,,off-target evidence,off-target evidence,Antisense oligonucleotide treatment produces a type I interferon response that protects against diet-induced obesity.,source:2770|molecule:1002,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,32180554,10.1016/j.molmet.2020.01.010
offtarget_safety_v0_1,train,offtarget_evidence,217,offtarget,A,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models.,source:12621|molecule:986,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,30114557,10.1016/j.omtn.2018.07.010
offtarget_safety_v0_1,train,offtarget_evidence,220,offtarget,A,unspecified siRNA (PMID:27461380),ASO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Ribonuclease H1-dependent hepatotoxicity caused by locked nucleic acid-modified gapmer antisense oligonucleotides.,source:2061|molecule:1003,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,27461380,10.1038/srep30377
offtarget_safety_v0_1,train,offtarget_evidence,331,offtarget,B,F7-1 siRNA,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Single base mismatches in the mRNA target site allow specific seed region-mediated off-target binding of siRNA targeting human coagulation factor 7.,source:15845|molecule:778,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,22258146,10.4161/rna.9.1.18121
offtarget_safety_v0_1,train,offtarget_evidence,333,offtarget,B,ATXN3-AON,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Determining off-target effects of splice-switching antisense oligonucleotides using short read RNAseq in neuronally differentiated human induced pluripotent stem cells.,source:1442|molecule:780,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,41066632,10.1093/hmg/ddaf153
offtarget_safety_v0_1,train,offtarget_evidence,334,offtarget,B,modified duplex,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Acyclic serinol nucleic acid modification of siRNAs overcomes seed region mediated off-target effects while maintaining potency.,source:1031|molecule:781,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,41994868,10.1093/nar/gkag345
offtarget_safety_v0_1,train,offtarget_evidence,337,offtarget,B,UNA-T siRNA,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs.,source:15362|molecule:782,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,21047800,10.1093/nar/gkq961
offtarget_safety_v0_1,train,offtarget_evidence,338,offtarget,B,siCD46 siRNA,siRNA,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Analysis of siRNA specificity on targets with double-nucleotide mismatches.,source:15336|molecule:783,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,18420656,10.1093/nar/gkn190
offtarget_safety_v0_1,train,offtarget_evidence,339,offtarget,B,BTK siRNA,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,siRNA-mediated off-target gene silencing triggered by a 7 nt complementation.,source:20894|molecule:784,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,16091630,10.1093/nar/gki762
offtarget_safety_v0_1,train,offtarget_evidence,340,offtarget,B,GR18-3 ASO,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Reduction of Off-Target Effects of Gapmer Antisense Oligonucleotides by Oligonucleotide Extension.,source:28684|molecule:785,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,34994962,10.1007/s40291-021-00573-z
offtarget_safety_v0_1,train,offtarget_evidence,343,offtarget,B,SOD-1,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Defining the factors that contribute to on-target specificity of antisense oligonucleotides.,source:31422|molecule:788,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,25072142,10.1371/journal.pone.0101752
offtarget_safety_v0_1,train,offtarget_evidence,345,offtarget,B,siRNA-5,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity.,source:2058|molecule:790,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,29459660,10.1038/s41467-018-02989-4
offtarget_safety_v0_1,train,offtarget_evidence,351,offtarget,B,siVIM-270,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,An siRMSD parameter of structural distortion induced by chemical modification is predictive of the off-target effect of siRNA.,source:1157|molecule:796,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,41467115,10.1016/j.omtn.2025.102693
offtarget_safety_v0_1,train,offtarget_evidence,355,offtarget,B,DM1,ASO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1.,source:1481|molecule:800,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,37200862,10.1016/j.omtn.2023.04.010
offtarget_safety_v0_1,train,offtarget_evidence,364,offtarget,B,siCdc2,siRNA,,,off-target evidence,off-target evidence,Site-Specific Modification Using the 2'-Methoxyethyl Group Improves the Specificity and Activity of siRNAs.,source:4230|molecule:809,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,29246303,10.1016/j.omtn.2017.10.003
offtarget_safety_v0_1,train,offtarget_evidence,370,offtarget,B,60 siRNA,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,siPools: highly complex but accurately defined siRNA pools eliminate off-target effects.,source:16582|molecule:815,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,24875475,10.1093/nar/gku480
offtarget_safety_v0_1,train,offtarget_evidence,383,offtarget,B,siEGFP1,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,A screen of chemical modifications identifies position-specific modification by UNA to most potently reduce siRNA off-target effects.,source:15558|molecule:826,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,20453030,10.1093/nar/gkq341
offtarget_safety_v0_1,train,offtarget_evidence,384,offtarget,B,Ago2; Ago1,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Off-target and a portion of target-specific siRNA mediated mRNA degradation is Ago2 'Slicer' independent and can be mediated by Ago1.,source:15470|molecule:827,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,19767612,10.1093/nar/gkp735
offtarget_safety_v0_1,train,offtarget_evidence,386,offtarget,B,TNFR1,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,Knockdown of TNFR1 by the sense strand of an ICAM-1 siRNA: dissection of an off-target effect.,source:21536|molecule:829,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,18096615,10.1093/nar/gkm630
offtarget_safety_v0_1,train,offtarget_evidence,390,offtarget,B,TS1-ASO,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Identification of nucleobase chemical modifications that reduce the hepatotoxicity of gapmer antisense oligonucleotides.,source:1409|molecule:833,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,35801870,10.1093/nar/gkac562
offtarget_safety_v0_1,train,offtarget_evidence,391,offtarget,B,S10-1,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects.,source:2253|molecule:834,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,36511872,10.1093/nar/gkac1108
offtarget_safety_v0_1,train,offtarget_evidence,392,offtarget,B,LNA ASO,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts.,source:2062|molecule:438,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,26553810,10.1093/nar/gkv1210
offtarget_safety_v0_1,train,offtarget_evidence,394,offtarget,B,TMEM16a,ASO,,,off-target evidence,off-target evidence,Restoring chloride efflux in cystic fibrosis with TMEM16a antisense oligonucleotides.,source:1233|molecule:836,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,40926414,10.1016/j.ymthe.2025.08.045
offtarget_safety_v0_1,train,offtarget_evidence,411,offtarget,B,PS-ASO,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Acute hepatotoxicity of 2' fluoro-modified 5-10-5 gapmer phosphorothioate oligonucleotides in mice correlates with intracellular protein binding and the loss of DBHS proteins.,source:2060|molecule:629,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,29390093,10.1093/nar/gky060
offtarget_safety_v0_1,train,offtarget_evidence,415,offtarget,B,WRN-108,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Development of an exon 27-skipping antisense oligonucleotide as a targeted therapy for refractory skin ulcers in Werner syndrome.,source:1055|molecule:851,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,42095135,10.1016/j.omtn.2026.102930
offtarget_safety_v0_1,train,offtarget_evidence,416,offtarget,B,KCNT1,ASO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy.,source:1058|molecule:852,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,41981306,10.1038/s41591-026-04314-9
offtarget_safety_v0_1,train,offtarget_evidence,417,offtarget,B,HIV-1,ASO/siRNA mixed context,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Investigation of the activity of phosphothioate and phosphothioate-LNA-modified oligonucleotides against HIV-1.,source:1210|molecule:853,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,41704705,10.3389/fmed.2026.1719202
offtarget_safety_v0_1,train,offtarget_evidence,436,offtarget,B,ASO-001933,ASO,,,seed-mediated off-target effect,seed-mediated off-target effect,Identification and characterization of a MAPT-targeting locked nucleic acid antisense oligonucleotide therapeutic for tauopathies.,source:1615|molecule:869,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,36090761,10.1016/j.omtn.2022.07.027
offtarget_safety_v0_1,train,offtarget_evidence,441,offtarget,B,T39 ASO,ASO,,,off-target evidence,off-target evidence,Antisense oligonucleotide treatment produces a type I interferon response that protects against diet-induced obesity.,source:2770|molecule:873,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,32180554,10.1016/j.molmet.2020.01.010
offtarget_safety_v0_1,train,offtarget_evidence,456,offtarget,B,modified siRNA,siRNA,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment.,source:3301|molecule:886,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,41552385,10.1016/j.omtn.2025.102802
offtarget_safety_v0_1,train,offtarget_evidence,463,offtarget,B,YAP1; WWTR1,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,A novel bispecific siRNA concept: Efficient dual knockdown of YAP1 and WWTR1 with a single guide strand.,source:1789|molecule:893,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,41341748,10.1016/j.omtn.2025.102768
offtarget_safety_v0_1,train,offtarget_evidence,464,offtarget,B,ACTB ASO,ASO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Targeted RNA editing by direct delivery of an adenosine deaminase-antisense oligo conjugate.,source:1824|molecule:894,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,40672232,10.1101/2025.07.11.664364
offtarget_safety_v0_1,train,offtarget_evidence,473,offtarget,B,UU-3,siRNA,,,off-target evidence,off-target evidence,Development of conditional-siRNA programmable riboswitch for targeting adverse cardiac remodeling.,source:3544|molecule:902,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,40861898,10.1016/j.omtn.2025.102667
offtarget_safety_v0_1,train,offtarget_evidence,493,offtarget,B,PH-762,siRNA,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Self-delivering RNAi immunotherapeutic PH-762 silences PD-1 to generate local and abscopal antitumor efficacy.,source:10539|molecule:921,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,39697325,10.3389/fimmu.2024.1501679
offtarget_safety_v0_1,train,offtarget_evidence,506,offtarget,B,siIGF1R-1,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Modified ASO conjugates encapsulated with cytidinyl/cationic lipids exhibit more potent and longer-lasting anti-HCC effects.,source:14112|molecule:932,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,37251692,10.1016/j.omtn.2023.04.028
offtarget_safety_v0_1,train,offtarget_evidence,510,offtarget,B,SLN360,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,"Preclinical Toxicological Assessment of A Novel siRNA, SLN360, Targeting Elevated Lipoprotein (a) in Cardiovascular Disease.",source:1486|molecule:187,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,35737426,10.1093/toxsci/kfac067
offtarget_safety_v0_1,train,offtarget_evidence,526,offtarget,B,HTT siRNA,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.,source:4213|molecule:881,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,31375812,10.1038/s41587-019-0205-0
offtarget_safety_v0_1,train,offtarget_evidence,530,offtarget,B,CEP290; LCA10,ASO/siRNA mixed context,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models.,source:12621|molecule:952,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,30114557,10.1016/j.omtn.2018.07.010
offtarget_safety_v0_1,train,offtarget_evidence,534,offtarget,B,89E-06,ASO,,,hybridization/mismatch off-target effect,hybridization/mismatch off-target effect,Strategies for In Vivo Screening and Mitigation of Hepatotoxicity Associated with Antisense Drugs.,source:2064|molecule:956,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,28918038,10.1016/j.omtn.2017.07.003
offtarget_safety_v0_1,validation,offtarget_evidence,82,offtarget,A,"siRNA-IGF2BP2 (recovered B2 from PMID:41859644, curator:CM)",siRNA,,,transcriptome-level off-target effect,transcriptome-level off-target effect,An exosome-biomimetic photothermal nanocarrier for IGF2BP2 siRNA delivery and enhanced ferroptosis in head and neck squamous cell carcinoma.,source:1732|molecule:993,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41859644,10.1016/j.mtbio.2026.102999
offtarget_safety_v0_1,validation,offtarget_evidence,157,offtarget,A,"Anti-N1 (recovered B2 from PMID:38379891, curator:CM)",ASO,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene.,source:1592|molecule:997,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38379891,10.1177/26331055241233596
offtarget_safety_v0_1,validation,offtarget_evidence,164,offtarget,A,"PH-762 (recovered B2 from PMID:39697325, curator:CM)",siRNA,,,transcriptome-level off-target effect,transcriptome-level off-target effect,Self-delivering RNAi immunotherapeutic PH-762 silences PD-1 to generate local and abscopal antitumor efficacy.,source:10539|molecule:998,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39697325,10.3389/fimmu.2024.1501679
offtarget_safety_v0_1,validation,offtarget_evidence,414,offtarget,B,CD-1,ASO,,,off-target evidence,off-target evidence,Sequence motifs associated with hepatotoxicity of locked nucleic acid--modified antisense oligonucleotides.,source:2063|molecule:850,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,24550163,10.1093/nar/gku142
offtarget_safety_v0_1,validation,offtarget_evidence,462,offtarget,B,si098-DV29P,siRNA,,,seed-mediated off-target effect,seed-mediated off-target effect,"Design, pharmacology, and toxicology of a novel chemically modified siRNA targeting hepatic angiotensinogen.",source:1477|molecule:892,source_plus_molecule_grouped_hash_v1,release_scale_review_batch012_fulltext_merged_manual_benchmark_v1,40385635,10.1016/j.omtn.2025.102542
toxicity_safety_v0_1,test,toxicity_endpoint,243,toxicity,B,unspecified antisense oligonucleotide,ASO,,,hepatic,hepatotoxicity,Hepatotoxic potential of therapeutic oligonucleotides can be predicted from their sequence and modification pattern.,source:23952551|molecule:unspecified antisense oligonucleotide,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,23952551,10.1089/nat.2013.0436
toxicity_safety_v0_1,test,toxicity_endpoint,416,toxicity,A,Nusinersen,ASO,SMN2,,general safety,adverse event reporting,Unveiling the adverse events of Nusinersen in spinal muscular atrophy management based on FAERS database.,source:39060346|molecule:Nusinersen,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,39060346,10.1038/s41598-024-67627-0
toxicity_safety_v0_1,test,toxicity_endpoint,418,toxicity,B,AVI-6002/AVI-6003 PMOplus,PMOplus,,,general safety,clinical safety and pharmacokinetics,Safety and pharmacokinetic profiles of phosphorodiamidate morpholino oligomers with activity against ebola virus and marburg virus: results of two single-ascending-dose studies.,source:25155593|molecule:AVI-6002/AVI-6003 PMOplus,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,25155593,10.1128/AAC.03442-14
toxicity_safety_v0_1,test,toxicity_endpoint,448,toxicity,B,P-HyOMe-WRAP5:siF3 nanoparticles,siRNA,FADD,,general safety,cell viability/cytotoxicity,Enhancing WRAP-Based Nanoparticles for Small Interfering Ribonucleic Acid Delivery in pH-Sensitive Environments.,source:9935|molecule:404,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,40084851,10.1002/cmdc.202400885
toxicity_safety_v0_1,test,toxicity_endpoint,449,toxicity,A,BE-ST@siBACE1 / BE-ST@siRNA,siRNA,BACE1,,immunotoxicity,immune activation,Berberine-inspired ionizable lipid for self-structure stabilization and brain targeting delivery of nucleic acid therapeutics.,source:9961|molecule:405,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,40064874,10.1038/s41467-025-57488-0
toxicity_safety_v0_1,test,toxicity_endpoint,453,toxicity,A,Ube3a-AS ASO,ASO,Ube3a-AS,,hepatic,hepatotoxicity,Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.,source:2614|molecule:409,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,38327047,10.1016/j.ymthe.2024.02.004
toxicity_safety_v0_1,test,toxicity_endpoint,460,toxicity,B,r/si-ORF7 flexible nano-liposomes,siRNA,ORF7,,general safety,cell viability/cytotoxicity,A novel recombinant ORF7-siRNA delivered by flexible nano-liposomes inhibits varicella zoster virus infection.,source:12511|molecule:416,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,37700336,10.1186/s13578-023-01108-1
toxicity_safety_v0_1,test,toxicity_endpoint,463,toxicity,A,CS-OA coated PLGA siRNA nanoparticles,siRNA,,,general safety,cell viability/cytotoxicity,Chitosan Oleate Coated PLGA Nanoparticles as siRNA Drug Delivery System.,source:4114|molecule:419,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,34684009,10.3390/pharmaceutics13101716
toxicity_safety_v0_1,test,toxicity_endpoint,529,toxicity,A,"D-siRNA (recovered B2 from PMID:41473322, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Albumin-binding dendrimer-conjugated siRNA enables safe and effective gene silencing throughout the central nervous system.,source:3402|molecule:1014,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41473322,10.64898/2025.12.16.694641
toxicity_safety_v0_1,test,toxicity_endpoint,540,toxicity,A,"L2-siRNA (recovered B2 from PMID:40598893, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid-siRNA conjugate administered into cerebrospinal fluid.,source:3619|molecule:1016,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40598893,10.1093/nar/gkaf600
toxicity_safety_v0_1,test,toxicity_endpoint,578,toxicity,A,"DG9-PMO (recovered B2 from PMID:40368879, curator:CM)",PMO,,,hepatic,hepatotoxicity,DG9 boosts PMO nuclear uptake and exon skipping to restore dystrophic muscle and cardiac function.,source:9560|molecule:1021,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40368879,10.1038/s41467-025-59494-8
toxicity_safety_v0_1,test,toxicity_endpoint,607,toxicity,A,"aODN-Survivin (recovered B2 from PMID:38672408, curator:CM)",ASO,,,hepatic,hepatotoxicity,Synthesis and Validation of TRIFAPYs as a Family of Transfection Agents for Therapeutic Oligonucleotides.,source:3903|molecule:1026,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38672408,10.3390/biom14040390
toxicity_safety_v0_1,test,toxicity_endpoint,616,toxicity,A,nusinersen,ASO,,,hepatic,hepatotoxicity,Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait.,source:11036|molecule:299,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38921951,10.3390/neurolint16030047
toxicity_safety_v0_1,test,toxicity_endpoint,634,toxicity,B,"siIRF1 (recovered B2 from PMID:39629104, curator:CM)",siRNA,,,general safety,cell viability/cytotoxicity,T Lymphocyte-Macrophage Hybrid Membrane-Coated Biomimetic Nanoparticles Alleviate Myocarditis via Suppressing Pyroptosis by Targeting Gene Silencing.,source:13678|molecule:1075,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39629104,10.2147/IJN.S487598
toxicity_safety_v0_1,test,toxicity_endpoint,664,toxicity,A,"LNP-S60-siRNA (recovered B2 from PMID:36520934, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Inhibition of c-Rel expression in myeloid and lymphoid cells with distearoyl -phosphatidylserine (DSPS) liposomal nanoparticles encapsulating therapeutic siRNA.,source:1964|molecule:1040,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,36520934,10.1371/journal.pone.0276905
toxicity_safety_v0_1,test,toxicity_endpoint,670,toxicity,A,unspecified siRNA (PMID:36457549),siRNA,,,hepatic,hepatotoxicity,Combined Self-Assembled iRGD Polymersomes for Effective Targeted siRNA Anti-Tumor Therapy.,source:4051|molecule:1043,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,36457549,10.2147/IJN.S383862
toxicity_safety_v0_1,test,toxicity_endpoint,679,toxicity,A,nusinersen,ASO,,,hepatic,hepatotoxicity,Reconditioning the Neurogenic Niche of Adult Non-human Primates by Antisense Oligonucleotide-Mediated Attenuation of TGFβ Signaling.,source:1645|molecule:299,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,33860461,10.1007/s13311-021-01045-2
toxicity_safety_v0_1,test,toxicity_endpoint,693,toxicity,A,Site-specific,ASO,,,hepatic,hepatotoxicity,Site-specific incorporation of 5'-methyl DNA enhances the therapeutic profile of gapmer ASOs.,source:14905|molecule:563,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,33544849,10.1093/nar/gkab047
toxicity_safety_v0_1,test,toxicity_endpoint,694,toxicity,A,unspecified PMO (PMID:33337582),PMO,,,hepatic,hepatotoxicity,MOTS-c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice.,source:14926|molecule:1046,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,33337582,10.15252/emmm.202012993
toxicity_safety_v0_1,test,toxicity_endpoint,717,toxicity,B,"revusiran (recovered B2 from PMID:28129130, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate.,source:2323|molecule:1077,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,28129130,10.1016/j.ymthe.2016.10.019
toxicity_safety_v0_1,test,toxicity_endpoint,727,toxicity,A,"LNA-i-miR-221 (recovered B2 from PMID:27327137, curator:CM)",miRNA agomir,,,hepatic,hepatotoxicity,Pharmacokinetics and Pharmacodynamics of a 13-mer LNA-inhibitor-miR-221 in Mice and Non-human Primates.,source:4239|molecule:1053,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,27327137,10.1038/mtna.2016.36
toxicity_safety_v0_1,test,toxicity_endpoint,1060,toxicity,A,Single-dose,siRNA,,,general safety,systemic safety/tolerability,Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model.,source:3717|molecule:652,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40196492,10.1101/2025.03.26.645328
toxicity_safety_v0_1,train,toxicity_endpoint,1,toxicity,A,phosphorothioate ASO panel from PMID 41760661,ASO,,,genotoxicity,DNA damage response,Dysregulation of the DNA damage response by phosphorothioate antisense oligonucleotides.,source:41760661|molecule:phosphorothioate ASO panel from PMID 41760661,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41760661,10.1038/s41467-026-69980-2
toxicity_safety_v0_1,train,toxicity_endpoint,2,toxicity,A,tool ASO safety panel from PMID 41918821,ASO,,,hepatic,hepatotoxicity,Integrating transcriptomics and proteomics to assess antisense oligonucleotide safety and efficacy: a time-resolved approach.,source:41918821|molecule:tool ASO safety panel from PMID 41918821,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41918821,10.1093/narmme/ugag016
toxicity_safety_v0_1,train,toxicity_endpoint,4,toxicity,A,5?-CP-modified PS-ASO panel from PMID 41467114,ASO,,,neurological,neurotoxicity,Unraveling and controlling late-onset neurotoxicity of antisense oligonucleotides through strategic chemical modifications.,source:41467114|molecule:17,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41467114,10.1016/j.omtn.2025.102692
toxicity_safety_v0_1,train,toxicity_endpoint,5,toxicity,B,BND6482,ASO,,,hepatic,hepatotoxicity,IGF1R-targeted delivery of a bridged nucleic acid oligonucleotide-peptide conjugate for microRNA-21 inhibition in triple-negative breast cancer.,source:41480638|molecule:BND6482,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41480638,10.1093/narcan/zcaf053
toxicity_safety_v0_1,train,toxicity_endpoint,6,toxicity,B,L2-Htt,siRNA,HTT,,general safety,safety/tolerability,Evaluating the effects of aging on biodistribution and gene silencing activity of lipid-siRNA conjugates delivered into cerebrospinal fluid.,source:41446212|molecule:L2-Htt,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41446212,10.64898/2025.12.17.693452
toxicity_safety_v0_1,train,toxicity_endpoint,8,toxicity,B,anti-miR-21-SNA,ASO,MIR21,,hepatic,hepatotoxicity,Ameliorative Effect of an Anti-MicroRNA-21 Oligonucleotide on Animal and Human Models of Cystic Kidney Disease.,source:40178922|molecule:anti-miR-21-SNA,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40178922,10.34067/KID.0000000771
toxicity_safety_v0_1,train,toxicity_endpoint,9,toxicity,A,LEM-S401,siRNA,CTGF,,general safety,safety/tolerability,"A First-In-Human Phase 1 Study to Evaluate the Safety and Tolerability of LEM-S401, a Novel siRNA-DegradaBALL Drug Targeting CTGF in Healthy Adults.",source:40160160|molecule:LEM-S401,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40160160,10.1111/cts.70207
toxicity_safety_v0_1,train,toxicity_endpoint,10,toxicity,A,hypoxanthine-substituted LNA-gapmer ASO1 panel from PMID 41928909,ASO,Mapt,,neurological,neurotoxicity,Guanine base modifications in antisense oligonucleotides mitigate acute central nervous system toxicity.,source:41928909|molecule:hypoxanthine-substituted LNA-gapmer ASO1 panel from PMID 41928909,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41928909,10.1039/d5cb00316d
toxicity_safety_v0_1,train,toxicity_endpoint,12,toxicity,A,RGLS4326 anti-miR-17 ASO,ASO,MIR17,,neurological,neurotoxicity,The nucleobase guanine at the 3'-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity.,source:41315228|molecule:RGLS4326 anti-miR-17 ASO,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41315228,10.1038/s41467-025-65799-5
toxicity_safety_v0_1,train,toxicity_endpoint,13,toxicity,A,SNS812,siRNA,SARS-CoV-2 RdRP,,general safety,safety/tolerability,"Pharmacokinetics and Safety Profile of SNS812, a First in Human Fully Modified siRNA Targeting Wide-Spectrum SARS-CoV-2, in Healthy Subjects.",source:40116355|molecule:SNS812,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40116355,10.1111/cts.70202
toxicity_safety_v0_1,train,toxicity_endpoint,16,toxicity,B,JNJ-3989/JNJ-73763989 GalNAc-siRNA regimen from PMID 40919077,siRNA,HBV,,general safety,safety/tolerability,Peginterferon-alpha-2a add-on to treatment with siRNA JNJ-73763989 in virologically suppressed chronic hepatitis B: The phase II PENGUIN study.,source:40919077|molecule:JNJ-3989/JNJ-73763989 GalNAc-siRNA regimen from PMID 40919077,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40919077,10.1016/j.jhepr.2025.101516
toxicity_safety_v0_1,train,toxicity_endpoint,17,toxicity,B,scFv-MML@LPCAT1si from PMID 40898177,siRNA,LPCAT1,,general safety,systemic safety,Biomimetic nanoparticles coated with ScFv-modified macrophage membranes for siRNA delivery to relieve brain metastases of lung cancer.,source:40898177|molecule:scFv-MML@LPCAT1si from PMID 40898177,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40898177,10.1186/s12896-025-01000-5
toxicity_safety_v0_1,train,toxicity_endpoint,19,toxicity,B,EM/siKEAP1@Fe-MOF@HA from PMID 40688667,siRNA,KEAP1,,general safety,systemic safety,Hyaluronic acid-anchored nanoparticles co-delivering emodin and siRNA confers protection against rheumatoid arthritis via macrophage polarization.,source:40688667|molecule:EM/siKEAP1@Fe-MOF@HA from PMID 40688667,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40688667,10.1016/j.mtbio.2025.102074
toxicity_safety_v0_1,train,toxicity_endpoint,21,toxicity,B,hEV@FAP-siTGFbeta1 from PMID 40046817,siRNA,TGFB1,,general safety,systemic safety,Injured Cardiac Tissue-Targeted Delivery of TGFβ1 siRNA by FAP Aptamer-Functionalized Extracellular Vesicles Promotes Cardiac Repair.,source:40046817|molecule:hEV@FAP-siTGFbeta1 from PMID 40046817,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40046817,10.2147/IJN.S497428
toxicity_safety_v0_1,train,toxicity_endpoint,22,toxicity,B,siAURKA-loaded milk extracellular vesicles from PMID 40896585,siRNA,AURKA,,immunotoxicity,immune activation,RNAi delivery mediated by milk extracellular vesicles in colon cancer.,source:40896585|molecule:siAURKA-loaded milk extracellular vesicles from PMID 40896585,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40896585,10.1016/j.omtn.2025.102644
toxicity_safety_v0_1,train,toxicity_endpoint,23,toxicity,B,BSCgal gold-siRNA supracluster from PMID 39448881,siRNA,LGALS1,,general safety,systemic safety,Gold-siRNA supraclusters enhance the anti-tumor immune response of stereotactic ablative radiotherapy at primary and metastatic tumors.,source:39448881|molecule:BSCgal gold-siRNA supracluster from PMID 39448881,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,39448881,10.1038/s41587-024-02448-0
toxicity_safety_v0_1,train,toxicity_endpoint,24,toxicity,A,G-clamp/BNAP-AEO modified Malat1 ASO panel from PMID 38978695,ASO,MALAT1,,neurological,neurotoxicity,"Favorable efficacy and reduced acute neurotoxicity by antisense oligonucleotides with 2',4'-BNA/LNA with 9-(aminoethoxy)phenoxazine.",source:38978695|molecule:G-clamp/BNAP-AEO modified Malat1 ASO panel from PMID 38978695,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,38978695,10.1016/j.omtn.2024.102161
toxicity_safety_v0_1,train,toxicity_endpoint,25,toxicity,A,aCSF+-formulated CNS ASO/di-siRNA panel from PMID 39554992,ASO/siRNA mixed context,,,neurological,neurotoxicity,Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca(2+) and Mg(2+) in the formulation.,source:39554992|molecule:aCSF+-formulated CNS ASO/di-siRNA panel from PMID 39554992,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,39554992,10.1016/j.omtn.2024.102359
toxicity_safety_v0_1,train,toxicity_endpoint,27,toxicity,B,LNP-siTGFbetaRI from PMID 39264964,siRNA,TGFBR1,,general safety,systemic safety,Development of a Lipid-encapsulated TGFβRI-siRNA Drug for Liver Fibrosis Induced by Schistosoma mansoni.,source:39264964|molecule:LNP-siTGFbetaRI from PMID 39264964,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,39264964,10.1371/journal.pntd.0012502
toxicity_safety_v0_1,train,toxicity_endpoint,28,toxicity,A,CT102,ASO,IGF1R,,general safety,safety/tolerability,Preclinical and phase I studies of an antisense oligonucleotide drug targeting IGF-1R in liver cancer.,source:38573183|molecule:CT102,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,38573183,10.2217/fon-2023-0872
toxicity_safety_v0_1,train,toxicity_endpoint,34,toxicity,B,OligoA-11/Skeen-11 phosphorothioate oligonucleotide panel from PMID 37185731,ASO,G6PD,,hepatic; renal,hepatic and renal toxicity,Inhibitory Effect of Phosphorothioate Oligonucleotide Complementary to G6PD mRNA on Murine Melanoma.,source:37185731|molecule:OligoA-11/Skeen-11 phosphorothioate oligonucleotide panel from PMID 37185731,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37185731,10.3390/cimb45040207
toxicity_safety_v0_1,train,toxicity_endpoint,35,toxicity,B,miR-125a-5p agomir from PMID 36632217,miRNA agomir,MIR125A,,general safety,cardiac and systemic safety,Therapeutic delivery of microRNA-125a-5p oligonucleotides improves recovery from myocardial ischemia/reperfusion injury in mice and swine.,source:36632217|molecule:miR-125a-5p agomir from PMID 36632217,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,36632217,10.7150/thno.73568
toxicity_safety_v0_1,train,toxicity_endpoint,36,toxicity,B,CLCN7G215R-siRNA-SiS12 formulation from PMID 37680985,siRNA,CLCN7,,general safety,systemic safety,Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. Implications for gene therapy in humans.,source:37680985|molecule:CLCN7G215R-siRNA-SiS12 formulation from PMID 37680985,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37680985,10.1016/j.omtn.2023.08.020
toxicity_safety_v0_1,train,toxicity_endpoint,37,toxicity,B,RBCEV-delivered siMstn and siMlycd from PMID 37016578,siRNA,MSTN; MLYCD,,immunotoxicity,immune and systemic safety,Red blood cell extracellular vesicles deliver therapeutic siRNAs to skeletal muscles for treatment of cancer cachexia.,source:37016578|molecule:RBCEV-delivered siMstn and siMlycd from PMID 37016578,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37016578,10.1016/j.ymthe.2023.03.036
toxicity_safety_v0_1,train,toxicity_endpoint,38,toxicity,A,ASO1-ASO3 modified ASO panel from PMID 36700050,ASO,,,neurological,neurotoxicity,Change of intracellular calcium level causes acute neurotoxicity by antisense oligonucleotides via CSF route.,source:36700050|molecule:ASO1-ASO3 modified ASO panel from PMID 36700050,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,36700050,10.1016/j.omtn.2022.12.010
toxicity_safety_v0_1,train,toxicity_endpoint,39,toxicity,A,TS1-ASO nucleobase modification panel from PMID 35801870,ASO,TTR,,hepatic,hepatotoxicity,Identification of nucleobase chemical modifications that reduce the hepatotoxicity of gapmer antisense oligonucleotides.,source:35801870|molecule:TS1-ASO nucleobase modification panel from PMID 35801870,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,35801870,10.1093/nar/gkac562
toxicity_safety_v0_1,train,toxicity_endpoint,40,toxicity,A,ESC+ GalNAc-siRNA D1/D4/D6/D9 panel from PMID 35736224,siRNA,TTR; HAO1,,hepatic,hepatotoxicity,From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization.,source:35736224|molecule:ESC+ GalNAc-siRNA D1/D4/D6/D9 panel from PMID 35736224,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,35736224,10.1093/nar/gkac539
toxicity_safety_v0_1,train,toxicity_endpoint,41,toxicity,B,DLin-MC3-DMA-conjugated siRNA panel from PMID 35917865,siRNA,HTT; PPIB,,general safety,transcriptome toxicity,Engineered ionizable lipid siRNA conjugates enhance endosomal escape but induce toxicity in vivo.,source:35917865|molecule:DLin-MC3-DMA-conjugated siRNA panel from PMID 35917865,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,35917865,10.1016/j.jconrel.2022.07.041
toxicity_safety_v0_1,train,toxicity_endpoint,42,toxicity,A,siLuc-ALC-0315 LNP from PMID 35642083,siRNA,LUC,,hepatic,hepatotoxicity,Comparison of DLin-MC3-DMA and ALC-0315 for siRNA Delivery to Hepatocytes and Hepatic Stellate Cells.,source:35642083|molecule:siLuc-ALC-0315 LNP from PMID 35642083,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,35642083,10.1021/acs.molpharmaceut.2c00033
toxicity_safety_v0_1,train,toxicity_endpoint,47,toxicity,B,CYP3A5 PMO/PSO panel from PMID 33633318,ASO,CYP3A5,,renal,renal safety,Antisense oligonucleotide development for the selective modulation of CYP3A5 in renal disease.,source:33633318|molecule:CYP3A5 PMO/PSO panel from PMID 33633318,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,33633318,10.1038/s41598-021-84194-w
toxicity_safety_v0_1,train,toxicity_endpoint,48,toxicity,A,HsPCSK9-1811-LNA/GalNAc ASO panel from PMID 34760338,ASO,PCSK9,,renal; hepatic,renal and hepatic safety,Drug discovery and development scheme for liver-targeting bridged nucleic acid antisense oligonucleotides.,source:34760338|molecule:HsPCSK9-1811-LNA/GalNAc ASO panel from PMID 34760338,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,34760338,10.1016/j.omtn.2021.10.008
toxicity_safety_v0_1,train,toxicity_endpoint,51,toxicity,B,LNPssPalmO-Phe-ASOap/ASOgr from PMID 33924589,ASO,APOB; NR3C1,,hepatic,hepatotoxicity,Delivery of Oligonucleotides Using a Self-Degradable Lipid-Like Material.,source:33924589|molecule:LNPssPalmO-Phe-ASOap/ASOgr from PMID 33924589,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,33924589,10.3390/pharmaceutics13040544
toxicity_safety_v0_1,train,toxicity_endpoint,54,toxicity,B,PMO-P6 conjugate from PMID 34841414,ASO,DMD,,renal,renal safety,Deep Learning Enables Discovery of a Short Nuclear Targeting Peptide for Efficient Delivery of Antisense Oligomers.,source:34841414|molecule:PMO-P6 conjugate from PMID 34841414,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,34841414,10.1021/jacsau.1c00327
toxicity_safety_v0_1,train,toxicity_endpoint,55,toxicity,B,PM1(GT/siMMP-9) hydrogel from PMID 33952251,siRNA,MMP9,,general safety,local and systemic safety,Sustained delivery of MMP-9 siRNA via thermosensitive hydrogel accelerates diabetic wound healing.,source:33952251|molecule:PM1(GT/siMMP-9) hydrogel from PMID 33952251,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,33952251,10.1186/s12951-021-00869-6
toxicity_safety_v0_1,train,toxicity_endpoint,56,toxicity,B,stereocontrolled cEt gapmer ASO panel from PMID 31980820,ASO,MALAT1,,general safety,cytotoxicity,Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides.,source:31980820|molecule:stereocontrolled cEt gapmer ASO panel from PMID 31980820,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,31980820,10.1093/nar/gkaa031
toxicity_safety_v0_1,train,toxicity_endpoint,58,toxicity,A,H51(+67)W tcDNA-ASO from PMID 31881528,ASO,DMD,,hepatic; renal,hepatic and renal safety,Identifying and Avoiding tcDNA-ASO Sequence-Specific Toxicity for the Development of DMD Exon 51 Skipping Therapy.,source:31881528|molecule:H51(+67)W tcDNA-ASO from PMID 31881528,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,31881528,10.1016/j.omtn.2019.11.020
toxicity_safety_v0_1,train,toxicity_endpoint,59,toxicity,A,GalNAc AON-B/D/E panel from PMID 30583097,ASO,,,renal,renal safety,GalNAc Conjugation Attenuates the Cytotoxicity of Antisense Oligonucleotide Drugs in Renal Tubular Cells.,source:30583097|molecule:GalNAc AON-B/D/E panel from PMID 30583097,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,30583097,10.1016/j.omtn.2018.11.005
toxicity_safety_v0_1,train,toxicity_endpoint,60,toxicity,A,GalNAc-siRNA hepatotoxicity panel with REVERSIR from PMID 29459660,siRNA,HAO1; TTR,,hepatic,hepatotoxicity,Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity.,source:29459660|molecule:GalNAc-siRNA hepatotoxicity panel with REVERSIR from PMID 29459660,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,29459660,10.1038/s41467-018-02989-4
toxicity_safety_v0_1,train,toxicity_endpoint,61,toxicity,A,236-LNA-ASO hepatotoxicity screening panel from PMID 29499955,ASO,,,hepatic,hepatotoxicity,A Sensitive In Vitro Approach to Assess the Hybridization-Dependent Toxic Potential of High Affinity Gapmer Oligonucleotides.,source:29499955|molecule:75,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,29499955,10.1016/j.omtn.2017.11.004
toxicity_safety_v0_1,train,toxicity_endpoint,62,toxicity,A,nephrotoxic AON A/B/C panel from PMID 28325303,ASO,,,renal,renal safety,Inhibition of EGF Uptake by Nephrotoxic Antisense Drugs In Vitro and Implications for Preclinical Safety Profiling.,source:28325303|molecule:nephrotoxic AON A/B/C panel from PMID 28325303,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,28325303,10.1016/j.omtn.2016.11.006
toxicity_safety_v0_1,train,toxicity_endpoint,63,toxicity,A,Acsl1/GR LNA gapmer panel from PMID 27461380,ASO,ACSL1; NR3C1,,hepatic,hepatotoxicity,Ribonuclease H1-dependent hepatotoxicity caused by locked nucleic acid-modified gapmer antisense oligonucleotides.,source:27461380|molecule:Acsl1/GR LNA gapmer panel from PMID 27461380,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,27461380,10.1038/srep30377
toxicity_safety_v0_1,train,toxicity_endpoint,64,toxicity,A,toxic LNA/MOE gapmer ASO panel from PMID 26553810,ASO,long pre-mRNA transcripts,,hepatic,hepatotoxicity,Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts.,source:26553810|molecule:toxic LNA/MOE gapmer ASO panel from PMID 26553810,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,26553810,10.1093/nar/gkv1210
toxicity_safety_v0_1,train,toxicity_endpoint,65,toxicity,B,PMO-GF formulation from PMID 26964641,ASO,DMD,,general safety,systemic safety,Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice.,source:26964641|molecule:PMO-GF formulation from PMID 26964641,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,26964641,10.1038/ncomms10981
toxicity_safety_v0_1,train,toxicity_endpoint,66,toxicity,A,3-8-3 LNA gapmer hepatotoxicity panel from PMID 24550163,ASO,,,hepatic,hepatotoxicity,Sequence motifs associated with hepatotoxicity of locked nucleic acid--modified antisense oligonucleotides.,source:24550163|molecule:80,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,24550163,10.1093/nar/gku142
toxicity_safety_v0_1,train,toxicity_endpoint,68,toxicity,A,siRNA candidate from PMID 41076269,siRNA,,,immunotoxicity,immune activation,Preclinical safety and bleeding evaluation in swine for a small interfering RNA-lipid nanoparticle that prevents excess fibrinogen synthesis.,source:41076269|molecule:siRNA candidate from PMID 41076269,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41076269,10.1016/j.jtha.2025.09.012
toxicity_safety_v0_1,train,toxicity_endpoint,69,toxicity,B,siRNA candidate from PMID 41716677,siRNA,,,immunotoxicity,immune activation,Spatiotemporal reprogramming of cardiac lipid metabolism by platelet-engineered RNA therapy epigenetically modulate heart repair and regeneration.,source:41716677|molecule:siRNA candidate from PMID 41716677,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41716677,10.1016/j.bioactmat.2026.02.009
toxicity_safety_v0_1,train,toxicity_endpoint,70,toxicity,A,siRNA candidate from PMID 40692538,siRNA,,,hematologic,platelet/bleeding safety,Protective Effects of GalNac-Modified Red Blood Cell-Derived Extracellular Vesicles Against Liver Diseases.,source:40692538|molecule:siRNA candidate from PMID 40692538,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40692538,10.2147/IJN.S510937
toxicity_safety_v0_1,train,toxicity_endpoint,71,toxicity,A,Plozasiran,ASO/siRNA mixed context,,,hematologic,platelet/bleeding safety,"Safety, pharmacokinetics and pharmacodynamics of Plozasiran in Chinese healthy volunteers.",source:41094680|molecule:Plozasiran,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41094680,10.1186/s12933-025-02929-9
toxicity_safety_v0_1,train,toxicity_endpoint,72,toxicity,B,siRNA candidate from PMID 40825982,siRNA,,,renal,renal safety,Targeting intestinal inflammation using locked nucleic acids delivered via lipid nanoparticles.,source:40825982|molecule:siRNA candidate from PMID 40825982,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40825982,10.1038/s41467-025-63037-6
toxicity_safety_v0_1,train,toxicity_endpoint,76,toxicity,B,siRNA candidate from PMID 40503175,siRNA,,,hepatic,hepatotoxicity,Targeting CIDEB alleviates liver steatosis and fibrosis in mouse MASH models.,source:40503175|molecule:siRNA candidate from PMID 40503175,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40503175,10.1016/j.omtn.2025.102567
toxicity_safety_v0_1,train,toxicity_endpoint,78,toxicity,A,Inclisiran,ASO/siRNA mixed context,,,hepatic,hepatotoxicity,Efficacy and Safety of Inclisiran in Asian Patients: Results From ORION-18.,source:38371290|molecule:Inclisiran,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,38371290,10.1016/j.jacasi.2023.09.006
toxicity_safety_v0_1,train,toxicity_endpoint,79,toxicity,A,ASO candidate from PMID 37365583,ASO,,,hematologic,platelet/bleeding safety,"Safety and activity of the first-in-class locked nucleic acid (LNA) miR-221 selective inhibitor in refractory advanced cancer patients: a first-in-human, phase 1, open-label, dose-escalation study.",source:37365583|molecule:ASO candidate from PMID 37365583,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37365583,10.1186/s13045-023-01468-8
toxicity_safety_v0_1,train,toxicity_endpoint,81,toxicity,B,ASO candidate from PMID 28853853,ASO,,,hepatic,hepatotoxicity,BNA(NC) Gapmers Revert Splicing and Reduce RNA Foci with Low Toxicity in Myotonic Dystrophy Cells.,source:28853853|molecule:ASO candidate from PMID 28853853,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,28853853,10.1021/acschembio.7b00416
toxicity_safety_v0_1,train,toxicity_endpoint,84,toxicity,A,Inotersen,ASO/siRNA mixed context,,,hematologic,platelet/bleeding safety,Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial.,source:32343462|molecule:Inotersen,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,32343462,10.1111/ene.14285
toxicity_safety_v0_1,train,toxicity_endpoint,85,toxicity,B,ASO candidate from PMID 41978261,ASO,,,immunotoxicity,immune activation,Phosphorothioate antisense oligonucleotide induced innate immune activation is attenuated by tryptophan oxidation products.,source:41978261|molecule:ASO candidate from PMID 41978261,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41978261,10.1093/nar/gkag311
toxicity_safety_v0_1,train,toxicity_endpoint,86,toxicity,B,SRN001,siRNA,,,hepatic,hepatotoxicity,"Safety and Pharmacokinetics of SRN001, a Novel siRNA Drug Targeting Amphiregulin via the SAMiRNA Platform.",source:42052104|molecule:SRN001,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,42052104,10.2147/DDDT.S589997
toxicity_safety_v0_1,train,toxicity_endpoint,88,toxicity,B,ASO candidate from PMID 39850319,ASO,,,renal,renal safety,In vivo efficacy and safety of systemically administered serinol nucleic acid-modified antisense oligonucleotides in mouse kidney.,source:39850319|molecule:ASO candidate from PMID 39850319,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,39850319,10.1016/j.omtn.2024.102387
toxicity_safety_v0_1,train,toxicity_endpoint,89,toxicity,B,ASO candidate from PMID 40243849,ASO,,,renal,renal safety,Inhibiting Myostatin Expression by the Antisense Oligonucleotides Improves Muscle Wasting in a Chronic Kidney Disease Mouse Model.,source:40243849|molecule:ASO candidate from PMID 40243849,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40243849,10.3390/ijms26073098
toxicity_safety_v0_1,train,toxicity_endpoint,90,toxicity,B,TLR9-targeted; STAT3; PD1-targeting ASO from PMID 40214214,ASO,,,immunotoxicity,immune activation,Multimodal glioma immunotherapy combining TLR9-targeted STAT3 antisense oligodeoxynucleotides with PD1 immune checkpoint blockade.,source:40214214|molecule:TLR9-targeted; STAT3; PD1-targeting ASO from PMID 40214214,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40214214,10.1093/neuonc/noaf099
toxicity_safety_v0_1,train,toxicity_endpoint,93,toxicity,B,siRNA candidate from PMID 41360814,siRNA,,,renal,renal safety,Inflammation-specific DNA origami nanodevice for delivery of siRNAs to treat ulcerative colitis.,source:41360814|molecule:siRNA candidate from PMID 41360814,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41360814,10.1038/s41467-025-67183-9
toxicity_safety_v0_1,train,toxicity_endpoint,94,toxicity,B,CD11b-modified-targeting siRNA from PMID 40977834,siRNA,,,renal,renal safety,CD11b-modified ROS/pH-responsive nanoparticles co-deliver Dioscin and siRNA to improve cardiac repair after myocardial infarction by reducing neutrophil recruitment.,source:40977834|molecule:CD11b-modified-targeting siRNA from PMID 40977834,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40977834,10.1016/j.mtbio.2025.102226
toxicity_safety_v0_1,train,toxicity_endpoint,95,toxicity,B,siRNA candidate from PMID 40380202,siRNA,,,immunotoxicity,immune activation,A novel pH-sensitive nanoparticles encapsulating anti-PD-1 antibody and MDK-siRNA overcome immune checkpoint blockade resistance in HCC via reshaping immunosuppressive TME.,source:40380202|molecule:siRNA candidate from PMID 40380202,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40380202,10.1186/s13046-025-03396-6
toxicity_safety_v0_1,train,toxicity_endpoint,96,toxicity,A,Angptl4-targeting ASO from PMID 39259836,ASO,,,immunotoxicity,immune activation,Liver-targeted Angptl4 silencing by antisense oligonucleotide treatment attenuates hyperlipidaemia and atherosclerosis development in APOE*3-Leiden.CETP mice.,source:39259836|molecule:Angptl4-targeting ASO from PMID 39259836,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,39259836,10.1093/cvr/cvae195
toxicity_safety_v0_1,train,toxicity_endpoint,97,toxicity,B,siRNA candidate from PMID 39606561,siRNA,,,renal,renal safety,A Multifunctional Nanodrug Co-Delivering VEGF-siRNA and Dexamethasone for Synergistic Therapy in Ocular Neovascular Diseases.,source:39606561|molecule:siRNA candidate from PMID 39606561,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,39606561,10.2147/IJN.S492363
toxicity_safety_v0_1,train,toxicity_endpoint,99,toxicity,B,siRNA candidate from PMID 38089931,siRNA,,,hematologic,platelet/bleeding safety,"Dendritic amphiphilic siRNA: Selective albumin binding, in vivo efficacy, and low toxicity.",source:38089931|molecule:siRNA candidate from PMID 38089931,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,38089931,10.1016/j.omtn.2023.102080
toxicity_safety_v0_1,train,toxicity_endpoint,100,toxicity,A,ASO candidate from PMID 36631935,ASO,,,general safety,safety/tolerability,Correlations between preclinical BJAB assay ranking of antisense drugs and clinical trial adverse events.,source:36631935|molecule:ASO candidate from PMID 36631935,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,36631935,10.1111/cts.13476
toxicity_safety_v0_1,train,toxicity_endpoint,101,toxicity,B,ASO candidate from PMID 37346977,ASO,,,neurological,neurotoxicity,Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection.,source:37346977|molecule:ASO candidate from PMID 37346977,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37346977,10.1016/j.omtn.2023.05.005
toxicity_safety_v0_1,train,toxicity_endpoint,102,toxicity,B,siRNA candidate from PMID 38168301,siRNA,,,renal,renal safety,Evaluation of mAb 2C5-modified dendrimer-based micelles for the co-delivery of siRNA and chemotherapeutic drug in xenograft mice model.,source:38168301|molecule:siRNA candidate from PMID 38168301,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,38168301,10.21203/rs.3.rs-3713164/v1
toxicity_safety_v0_1,train,toxicity_endpoint,105,toxicity,B,ASO candidate from PMID 33567808,ASO,,,hematologic,platelet/bleeding safety,"Sequence-specific 2'-O-methoxyethyl antisense oligonucleotides activate human platelets through glycoprotein VI, triggering formation of platelet-leukocyte aggregates.",source:33567808|molecule:ASO candidate from PMID 33567808,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,33567808,10.3324/haematol.2020.260059
toxicity_safety_v0_1,train,toxicity_endpoint,106,toxicity,B,siRNA candidate from PMID 35992044,siRNA,,,hepatic,hepatotoxicity,A model system for antiviral siRNA therapeutics using exosome-based delivery.,source:35992044|molecule:siRNA candidate from PMID 35992044,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,35992044,10.1016/j.omtn.2022.08.011
toxicity_safety_v0_1,train,toxicity_endpoint,107,toxicity,B,siRNA candidate from PMID 30544191,siRNA,,,renal,renal safety,Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo.,source:30544191|molecule:siRNA candidate from PMID 30544191,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,30544191,10.1093/nar/gky1239
toxicity_safety_v0_1,train,toxicity_endpoint,110,toxicity,A,Nedosiran,ASO/siRNA mixed context,,,renal,renal safety,"Nedosiran in pediatric patients with PH1 and relatively preserved kidney function, a phase 2 study (PHYOX8).",source:39875734|molecule:Nedosiran,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,39875734,10.1007/s00467-025-06675-8
toxicity_safety_v0_1,train,toxicity_endpoint,111,toxicity,A,Nedosiran,ASO/siRNA mixed context,,,renal,renal safety,Nedosiran Safety and Efficacy in PH1: Interim Analysis of PHYOX3.,source:38707801|molecule:Nedosiran,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,38707801,10.1016/j.ekir.2024.02.1439
toxicity_safety_v0_1,train,toxicity_endpoint,112,toxicity,B,DNA/RNA heteroduplex oligonucleotide candidate from PMID 39252874,DNA/RNA heteroduplex oligonucleotide,,,immunotoxicity,immune activation,DNA/RNA heteroduplex technology with cationic oligopeptide reduces class-related adverse effects of nucleic acid drugs.,source:39252874|molecule:DNA/RNA heteroduplex oligonucleotide candidate from PMID 39252874,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,39252874,10.1016/j.omtn.2024.102289
toxicity_safety_v0_1,train,toxicity_endpoint,113,toxicity,A,Viltolarsen,ASO,,,renal,renal safety,"Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.",source:37005891|molecule:Viltolarsen,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37005891,10.3233/JND-221656
toxicity_safety_v0_1,train,toxicity_endpoint,114,toxicity,A,Viltolarsen,ASO,,,general safety,safety/tolerability,Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.,source:35634851|molecule:Viltolarsen,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,35634851,10.3233/JND-220811
toxicity_safety_v0_1,train,toxicity_endpoint,116,toxicity,A,Inclisiran,ASO/siRNA mixed context,,,general safety,safety/tolerability,"Efficacy, Safety, and Tolerability of Inclisiran in Patients With Homozygous Familial Hypercholesterolemia: Results From the ORION-5 Randomized Clinical Trial.",source:37850379|molecule:Inclisiran,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37850379,10.1161/CIRCULATIONAHA.122.063460
toxicity_safety_v0_1,train,toxicity_endpoint,117,toxicity,B,siRNA candidate from PMID 41949334,siRNA,,,hepatic,hepatotoxicity,A Long-Term Human Liver Spheroid Model for Assessing Silencing and Durability of GalNAc-Conjugated siRNAs.,source:41949334|molecule:siRNA candidate from PMID 41949334,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41949334,10.1111/cts.70536
toxicity_safety_v0_1,train,toxicity_endpoint,136,toxicity,B,Fitusiran,ASO,,,hepatic,hepatotoxicity,"Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B.",source:39642315|molecule:Fitusiran,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,39642315,10.1182/bloodadvances.2024013900
toxicity_safety_v0_1,train,toxicity_endpoint,147,toxicity,A,Nusinersen,ASO/siRNA mixed context,,,hematologic,platelet/bleeding safety,Real-world analysis of the efficacy and safety of nusinersen in pediatric patients with spinal muscular atrophy.,source:40011938|molecule:Nusinersen,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40011938,10.1186/s13023-025-03603-9
toxicity_safety_v0_1,train,toxicity_endpoint,150,toxicity,A,Olezarsen,ASO/siRNA mixed context,,,hematologic,platelet/bleeding safety,Efficacy and safety of olezarsen in lowering apolipoprotein C-III and triglycerides in healthy Japanese Americans.,source:39363329|molecule:Olezarsen,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,39363329,10.1186/s12944-024-02297-5
toxicity_safety_v0_1,train,toxicity_endpoint,151,toxicity,A,ASO/siRNA mixed context candidate from PMID 38271438,ASO/siRNA mixed context,,,hepatic,hepatotoxicity,A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice.,source:38271438|molecule:ASO/siRNA mixed context candidate from PMID 38271438,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,38271438,10.1371/journal.pone.0294847
toxicity_safety_v0_1,train,toxicity_endpoint,154,toxicity,A,Inclisiran,ASO/siRNA mixed context,,,hepatic,hepatotoxicity,"Efficacy, Safety, and Pharmacokinetics of Inclisiran in Japanese Patients: Results from ORION-15.",source:38220186|molecule:Inclisiran,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,38220186,10.5551/jat.64454
toxicity_safety_v0_1,train,toxicity_endpoint,159,toxicity,B,ASO candidate from PMID 37920238,ASO,,,neurological,neurotoxicity,Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model.,source:37920238|molecule:ASO candidate from PMID 37920238,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37920238,10.1016/j.omtm.2023.101122
toxicity_safety_v0_1,train,toxicity_endpoint,163,toxicity,A,unspecified siRNA,siRNA,,,general safety,cell viability/cytotoxicity,A Straightforward Method for the Development of Positively Charged Gold Nanoparticle-Based Vectors for Effective siRNA Delivery.,source:37110552|molecule:unspecified siRNA,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37110552,10.3390/molecules28083318
toxicity_safety_v0_1,train,toxicity_endpoint,167,toxicity,A,inotersen,ASO/siRNA mixed context,,,hepatic,hepatotoxicity,Long-term efficacy and safety of inotersen for hereditary transthyretin amyloidosis: NEURO-TTR open-label extension 3-year update.,source:35908242|molecule:inotersen,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,35908242,10.1007/s00415-022-11276-8
toxicity_safety_v0_1,train,toxicity_endpoint,174,toxicity,A,HepG2; LO2,siRNA,,,hepatic,hepatotoxicity,cRGD peptide-conjugated polyethylenimine-based lipid nanoparticle for intracellular delivery of siRNA in hepatocarcinoma therapy.,source:34042551|molecule:HepG2; LO2,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,34042551,10.1080/10717544.2021.1928794
toxicity_safety_v0_1,train,toxicity_endpoint,176,toxicity,B,Foxp3,ASO,,,hepatic,hepatotoxicity,Foxp3 Silencing with Antisense Oligonucleotide Improves Immunogenicity of an Adjuvanted Recombinant Vaccine against Sporothrix schenckii.,source:33801683|molecule:Foxp3,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,33801683,10.3390/ijms22073470
toxicity_safety_v0_1,train,toxicity_endpoint,177,toxicity,B,Revusiran,siRNA,,,hepatic,hepatotoxicity,"Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis.",source:31821125|molecule:Revusiran,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,31821125,10.1089/nat.2019.0796
toxicity_safety_v0_1,train,toxicity_endpoint,181,toxicity,A,radavirsen,ASO,,,hepatic,hepatotoxicity,"Safety, tolerability, and pharmacokinetics of radavirsen (AVI-7100), an antisense oligonucleotide targeting influenza a M1/M2 translation.",source:28929521|molecule:radavirsen,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,28929521,10.1111/bcp.13405
toxicity_safety_v0_1,train,toxicity_endpoint,184,toxicity,B,unspecified antisense oligonucleotide,ASO,,,hepatic,hepatotoxicity,Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice.,source:28633548|molecule:unspecified antisense oligonucleotide,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,28633548,10.1080/10717544.2017.1337827
toxicity_safety_v0_1,train,toxicity_endpoint,188,toxicity,B,unspecified antisense oligonucleotide,ASO,,,hepatic,hepatotoxicity,Evaluation of Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked polyethylenimine as antisense morpholino oligomer delivery vehicle in cell culture and dystrophic mdx mice.,source:24405395|molecule:unspecified antisense oligonucleotide,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,24405395,10.1089/hum.2013.156
toxicity_safety_v0_1,train,toxicity_endpoint,192,toxicity,A,unspecified oligonucleotide,ASO/siRNA mixed context,,,renal,renal safety,"Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.",source:34788571|molecule:unspecified oligonucleotide,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,34788571,10.1089/nat.2021.0043
toxicity_safety_v0_1,train,toxicity_endpoint,194,toxicity,A,MC3-based,siRNA,,,hepatic,hepatotoxicity,Efficiency and safety of five different agents for in vivo delivery of novel bioengineered RNAi molecules.,source:41907143|molecule:MC3-based,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,41907143,10.3389/fmolb.2026.1785592
toxicity_safety_v0_1,train,toxicity_endpoint,209,toxicity,B,givosiran,siRNA,,,hepatic,hepatotoxicity,Efficacy and safety of givosiran in Japanese patients with acute hepatic porphyria: clinical findings from an expanded access study.,source:40312531|molecule:givosiran,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40312531,10.1038/s41598-025-99526-3
toxicity_safety_v0_1,train,toxicity_endpoint,210,toxicity,A,fitusiran,ASO/siRNA mixed context,,,hepatic,hepatotoxicity,Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study.,source:40053895|molecule:fitusiran,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,40053895,10.1182/blood.2024027008
toxicity_safety_v0_1,train,toxicity_endpoint,229,toxicity,A,inclisiran,ASO/siRNA mixed context,,,general safety,safety/tolerability,"Efficacy and safety of inclisiran in patients with cerebrovascular disease: ORION-9, ORION-10, and ORION-11.",source:37252442|molecule:inclisiran,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37252442,10.1016/j.ajpc.2023.100503
toxicity_safety_v0_1,train,toxicity_endpoint,230,toxicity,B,PF4,ASO,,,renal,renal safety,"Platelet Activation by Antisense Oligonucleotides (ASOs) in the Göttingen Minipig, including an Evaluation of Glycoprotein VI (GPVI) and Platelet Factor 4 (PF4) Ontogeny.",source:37111598|molecule:PF4,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,37111598,10.3390/pharmaceutics15041112
toxicity_safety_v0_1,train,toxicity_endpoint,235,toxicity,A,unspecified antisense oligonucleotide,ASO,,,hepatic,hepatotoxicity,Antisense oligonucleotides and nucleic acids generate hypersensitive platelets.,source:33540294|molecule:unspecified antisense oligonucleotide,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,33540294,10.1016/j.thromres.2021.01.006
toxicity_safety_v0_1,train,toxicity_endpoint,239,toxicity,A,unspecified antisense oligonucleotide,ASO,,,hepatic,hepatotoxicity,Intracerebroventricular Administration of a 2'-O-Methyl Phosphorothioate Antisense Oligonucleotide Results in Activation of the Innate Immune System in Mouse Brain.,source:29565739|molecule:unspecified antisense oligonucleotide,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,29565739,10.1089/nat.2017.0705
toxicity_safety_v0_1,train,toxicity_endpoint,244,toxicity,A,nusinersen,ASO/siRNA mixed context,,,general safety,safety/tolerability,Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study.,source:38361750|molecule:nusinersen,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,38361750,10.1016/j.lanepe.2024.100862
toxicity_safety_v0_1,train,toxicity_endpoint,414,toxicity,A,Inclisiran,siRNA,PCSK9,,general safety,clinical laboratory safety,"Efficacy and Safety of Inclisiran in Adolescents With Genetically Confirmed Homozygous Familial Hypercholesterolemia: Results From the Double-Blind, Placebo-Controlled Part of the ORION-13 Randomized Trial.",source:40391436|molecule:Inclisiran,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,40391436,10.1161/CIRCULATIONAHA.124.073233
toxicity_safety_v0_1,train,toxicity_endpoint,417,toxicity,A,Inclisiran,siRNA,PCSK9,,general safety,treatment-emergent adverse events,"Efficacy and Safety of Inclisiran in Patients with Polyvascular Disease: Pooled, Post Hoc Analysis of the ORION-9, ORION-10, and ORION-11 Phase 3 Randomized Controlled Trials.",source:36550348|molecule:Inclisiran,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,36550348,10.1007/s10557-022-07413-0
toxicity_safety_v0_1,train,toxicity_endpoint,419,toxicity,A,phosphorothioate ASO panel,ASO,,,neurological,acute neuronal inhibition,Acute neuronal inhibition response caused by phosphorothioate antisense oligonucleotides following local delivery to the central nervous system.,source:41494985|molecule:phosphorothioate ASO panel,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41494985,10.1093/nar/gkaf1333
toxicity_safety_v0_1,train,toxicity_endpoint,421,toxicity,A,TET3 siRNA lipid nanoparticles,siRNA,TET3,,general safety,hemolysis and cytotoxicity,Dual-targeted lipid nanoparticles for TET3 siRNA delivery: nanobiotechnology strategy to remodel tumor immune microenvironment in hepatocellular carcinoma.,source:41923247|molecule:TET3 siRNA lipid nanoparticles,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41923247,10.1186/s12951-026-04312-6
toxicity_safety_v0_1,train,toxicity_endpoint,424,toxicity,A,FW-LP@siIRF5,siRNA,IRF5,,general safety,hematology/clinical chemistry/histology safety,Reprogramming Lesional Macrophage Homeostasis via Interferon Regulatory Factor 5 Targeted siRNA Nanoimmunotherapy for Atherosclerosis.,source:41776767|molecule:FW-LP@siIRF5,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41776767,10.1021/acsnano.5c18044
toxicity_safety_v0_1,train,toxicity_endpoint,425,toxicity,A,DCA-JAK13033 siRNA,siRNA,JAK1,,general safety,systemic exposure and immunogenicity safety,Intradermal delivery of lipophilic siRNAs enables prolonged skin retention and sustained gene silencing in a porcine model.,source:41634015|molecule:DCA-JAK13033 siRNA,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41634015,10.1038/s41467-026-68993-1
toxicity_safety_v0_1,train,toxicity_endpoint,427,toxicity,A,DP-EVs/siPD-L1,siRNA,CD274,,general safety,systemic toxicity/histology/body weight,An Engineered Extracellular Vesicle With Enhanced Tumor and Lymph Nodes Targeting as siRNA Delivery System for Robust Tumor Immunotherapy.,source:41815210|molecule:DP-EVs/siPD-L1,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41815210,10.1002/mco2.70673
toxicity_safety_v0_1,train,toxicity_endpoint,436,toxicity,A,siRNA@VG-Exos,siRNA,IL6,,hepatic/renal/general safety,histology and serum biochemistry safety,Endothelial-targeted modification of ginseng-derived exosomes for IL-6 SiRNA delivery ameliorates hepatic ischemia-reperfusion injury.,source:41491215|molecule:siRNA@VG-Exos,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41491215,10.1186/s12951-025-03954-2
toxicity_safety_v0_1,train,toxicity_endpoint,437,toxicity,B,LNP-SNORA49 RNA/SOX9 ASO,ASO/RNA mixed context,SOX9,,general safety,organ morphology/body weight/cytokine safety,SNORA49 negatively regulates self-renewal of liver cancer stem cells and hepatocarcinogenesis via suppressing SOX9 transcription.,source:41565615|molecule:LNP-SNORA49 RNA/SOX9 ASO,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41565615,10.1038/s41467-025-66486-1
toxicity_safety_v0_1,train,toxicity_endpoint,439,toxicity,A,pMSNC@ISG15 siRNA,siRNA,ISG15,,general safety,in vivo biocompatibility/clinical chemistry,Interferon-stimulated gene 15 small interfering RNA-loaded polarized mesoporous silica nanocarriers remodel the immune microenvironment to ameliorate post-myocardial infarction heart failure.,source:41560785|molecule:pMSNC@ISG15 siRNA,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41560785,10.1016/j.mtbio.2025.102631
toxicity_safety_v0_1,train,toxicity_endpoint,440,toxicity,A,SANG-siRNA,siRNA,,,general safety,systemic toxicity/repeat-dose tolerability,Tumor agnostic drug delivery with dynamic nanohydrogels.,source:41500988|molecule:SANG-siRNA,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41500988,10.1038/s41467-025-66788-4
toxicity_safety_v0_1,train,toxicity_endpoint,441,toxicity,A,cationic/anionic siRNA lipoplexes,siRNA,,,general safety,cell viability/cytotoxicity,Assessment of anionic siRNA lipoplexes prepared via modified ethanol injection for tumor cell delivery.,source:41282509|molecule:cationic/anionic siRNA lipoplexes,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41282509,10.3892/br.2025.2085
toxicity_safety_v0_1,train,toxicity_endpoint,442,toxicity,B,ISIS 405879,ASO,,,hematological,thrombocytopenia,Cellular immune changes during severe antisense oligonucleotide-associated thrombocytopenia in a nonhuman primate model.,source:40101742|molecule:ISIS 405879,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,40101742,10.1093/jimmun/vkae055
toxicity_safety_v0_1,train,toxicity_endpoint,450,toxicity,A,PEI/PHD2 siRNA nanocomplexes,siRNA,PHD2,,general safety,cell viability/cytotoxicity,Optimized delivery of RNA silencing prolyl hydroxylase domain 2 for enhanced angiogenesis and osteogenesis using bioactive glass scaffolds.,source:12938|molecule:406,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,41266661,10.1038/s41598-025-24808-9
toxicity_safety_v0_1,train,toxicity_endpoint,451,toxicity,A,PLGA-CpG@ID8-M nanovaccine,CpG oligodeoxynucleotide,,,general safety,cell viability/cytotoxicity,CpG-Based Nanovaccines Enhance Ovarian Cancer Immune Response by Gbp2-Mediated Remodeling of Tumor-Associated Macrophages.,source:13359|molecule:407,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,39985265,10.1002/advs.202412881
toxicity_safety_v0_1,train,toxicity_endpoint,454,toxicity,A,CD44 siRNA nanoplexes,siRNA,CD44,,general safety,cell viability/cytotoxicity,Development of Focused Ultrasound-Assisted Nanoplexes for RNA Delivery.,source:10973|molecule:410,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,38998694,10.3390/nano14131089
toxicity_safety_v0_1,train,toxicity_endpoint,455,toxicity,A,Phe-NPs/ASO,ASO,,,general safety,cell viability/cytotoxicity,l-Type amino acid transporter 1-targeting nanoparticles for antisense oligonucleotide delivery to the CNS.,source:12161|molecule:411,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,39411247,10.1016/j.omtn.2024.102340
toxicity_safety_v0_1,train,toxicity_endpoint,456,toxicity,A,DOTAP/siSmad4@EM,siRNA,Smad4,,immunotoxicity,immune activation,Pulmonary fibroblast-specific delivery of siRNA exploiting exosomes-based nanoscaffolds for IPF treatment.,source:12475|molecule:412,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,39258001,10.1016/j.ajps.2024.100929
toxicity_safety_v0_1,train,toxicity_endpoint,457,toxicity,A,TPNs-siRNA,siRNA,MMP-2/MMP-9,,general safety,systemic safety/tolerability,Tea polyphenol nanoparticles enable targeted siRNA delivery and multi-bioactive therapy for abdominal aortic aneurysms.,source:12485|molecule:413,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,39118143,10.1186/s12951-024-02756-2
toxicity_safety_v0_1,train,toxicity_endpoint,459,toxicity,A,AA-T3A-C12/siHSP47 LNP,siRNA,HSP47,,hepatic,hepatotoxicity,Ligand-tethered lipid nanoparticles for targeted RNA delivery to treat liver fibrosis.,source:4035|molecule:415,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,36650129,10.1038/s41467-022-35637-z
toxicity_safety_v0_1,train,toxicity_endpoint,461,toxicity,A,MPLN siRNA nanoparticles,siRNA,,,general safety,cell viability/cytotoxicity,Lymphocyte Membrane- and 12p1-Dual-Functionalized Nanoparticles for Free HIV-1 Trapping and Precise siRNA Delivery into HIV-1-Infected Cells.,source:12533|molecule:417,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,36755201,10.1002/advs.202300282
toxicity_safety_v0_1,train,toxicity_endpoint,462,toxicity,A,ZNF416 siRNA-loaded liposomes,siRNA,ZNF416,,general safety,systemic safety/tolerability,Targeted delivery of ZNF416 siRNA-loaded liposomes attenuates experimental pulmonary fibrosis.,source:12545|molecule:418,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,36371191,10.1186/s12967-022-03740-w
toxicity_safety_v0_1,train,toxicity_endpoint,465,toxicity,A,siSTAT3/DoCh LNP,siRNA,STAT3,,hepatic,hepatotoxicity,Silencing of STAT3 via Peptidomimetic LNP-Mediated Systemic Delivery of RNAi Downregulates PD-L1 and Inhibits Melanoma Growth.,source:4197|molecule:421,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,32059541,10.3390/biom10020285
toxicity_safety_v0_1,train,toxicity_endpoint,470,toxicity,A,Br-ApoE(K->A)-PMO and related P-PMO conjugates,PMO,,,general safety,cell viability/cytotoxicity,Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy.,source:12658|molecule:426,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,28118087,10.1089/nat.2016.0652
toxicity_safety_v0_1,train,toxicity_endpoint,475,toxicity,A,unspecified ASO,ASO,,,hepatic,hepatotoxicity,Development of an exon 27-skipping antisense oligonucleotide as a targeted therapy for refractory skin ulcers in Werner syndrome.,source:1055|molecule:494,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,42095135,10.1016/j.omtn.2026.102930
toxicity_safety_v0_1,train,toxicity_endpoint,476,toxicity,A,unspecified ASO (PMID:41981306),ASO,,,general safety,systemic safety/tolerability,Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy.,source:1058|molecule:1005,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41981306,10.1038/s41591-026-04314-9
toxicity_safety_v0_1,train,toxicity_endpoint,478,toxicity,A,unspecified ASO (PMID:42033225),ASO,,,hepatic,hepatotoxicity,Direct targeting of C9ORF72 repeat RNA with fluorinated antisense oligonucleotides.,source:1185|molecule:1007,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,42033225,10.1093/nar/gkag343
toxicity_safety_v0_1,train,toxicity_endpoint,480,toxicity,A,CO2,ASO,,,general safety,cell viability/cytotoxicity,Investigation of the activity of phosphothioate and phosphothioate-LNA-modified oligonucleotides against HIV-1.,source:1210|molecule:508,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41704705,10.3389/fmed.2026.1719202
toxicity_safety_v0_1,train,toxicity_endpoint,481,toxicity,A,"siRNA2 (recovered B2 from PMID:42005705, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Preparation and Antitumor Activity of METTL3 SiRNA Nanoparticles Carried by Hypoxia Sensitive Polymer (mPEG-Azo-PEI).,source:1368|molecule:1008,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,42005705,10.2147/IJN.S588018
toxicity_safety_v0_1,train,toxicity_endpoint,483,toxicity,A,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic,hepatotoxicity,Silencing of PAI-1 using siRNA-lipid nanoparticles reduces thrombosis and prolongs life span in murine models.,source:2412|molecule:985,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41587091,10.1182/blood.2025029834
toxicity_safety_v0_1,train,toxicity_endpoint,485,toxicity,A,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic,hepatotoxicity,PARPi Combining Nanoparticle LIN28B siRNA for the Management of Malignant Ascites.,source:3294|molecule:985,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41572432,10.1002/advs.202510547
toxicity_safety_v0_1,train,toxicity_endpoint,492,toxicity,A,"eteplirsen (recovered B2 from PMID:41961051, curator:CM)",PMO,,,hepatic,hepatotoxicity,"Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD.",source:5375|molecule:1009,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41961051,10.57264/cer-2025-0108
toxicity_safety_v0_1,train,toxicity_endpoint,499,toxicity,A,tofersen,ASO,,,general safety,systemic safety/tolerability,Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis.,source:6390|molecule:514,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41661214,10.1001/jamaneurol.2025.4946
toxicity_safety_v0_1,train,toxicity_endpoint,500,toxicity,A,unspecified ASO (PMID:41630990),ASO,,,hepatic,hepatotoxicity,Improving angiogenesis ameliorates the efficacy of ASO-based exon skipping for the treatment of Duchenne muscular dystrophy.,source:6475|molecule:1010,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41630990,10.1016/j.omtn.2026.102834
toxicity_safety_v0_1,train,toxicity_endpoint,511,toxicity,A,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,general safety,cell viability/cytotoxicity,Genetically bio-engineered PD-L1 targeted exosomes for immunotherapy of resistant triple negative breast cancer.,source:7336|molecule:985,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40762902,10.1007/s13346-025-01920-x
toxicity_safety_v0_1,train,toxicity_endpoint,512,toxicity,B,"elsunersen (recovered B2 from PMID:40263630, curator:CM)",ASO,,,hepatic,hepatotoxicity,Antisense oligonucleotide treatment in a preterm infant with early-onset SCN2A developmental and epileptic encephalopathy.,source:1013|molecule:1066,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40263630,10.1038/s41591-025-03656-0
toxicity_safety_v0_1,train,toxicity_endpoint,513,toxicity,A,"ASO 8–3 (recovered B2 from PMID:40098305, curator:CM)",ASO,,,hepatic,hepatotoxicity,AmNA-Modified Antisense Oligonucleotide Targeting MCM8 as a Cancer-Specific Chemosensitizer for Platinum Compounds.,source:1020|molecule:1011,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40098305,10.1111/cas.70024
toxicity_safety_v0_1,train,toxicity_endpoint,514,toxicity,A,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic,hepatotoxicity,Restoring chloride efflux in cystic fibrosis with TMEM16a antisense oligonucleotides.,source:1233|molecule:986,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40926414,10.1016/j.ymthe.2025.08.045
toxicity_safety_v0_1,train,toxicity_endpoint,516,toxicity,B,single-patient,ASO,,,hepatic,hepatotoxicity,"Antisense oligonucleotide jacifusen for FUS-ALS: an investigator-initiated, multicentre, open-label case series.",source:1254|molecule:521,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40414239,10.1016/S0140-6736(25)00513-6
toxicity_safety_v0_1,train,toxicity_endpoint,517,toxicity,A,nusinersen,ASO,,,general safety,systemic safety/tolerability,Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy.,source:1256|molecule:299,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40367190,10.1126/scitranslmed.adv4656
toxicity_safety_v0_1,train,toxicity_endpoint,518,toxicity,A,AZD8701,ASO,,,hepatic,hepatotoxicity,"AZD8701, an Antisense Oligonucleotide Targeting FOXP3 mRNA, as Monotherapy and in Combination with Durvalumab: A Phase I Trial in Patients with Advanced Solid Tumors.",source:1270|molecule:522,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39937271,10.1158/1078-0432.CCR-24-1818
toxicity_safety_v0_1,train,toxicity_endpoint,520,toxicity,A,"Chol-HDO[PMO] (recovered B2 from PMID:41429785, curator:CM)",PMO,,,hepatic,hepatotoxicity,"Morpholino-RNA duplex exhibits robust, sustained, and safe steric-block antisense activity by intracerebroventricular and intrathecal injection.",source:1783|molecule:1012,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41429785,10.1038/s41467-025-66765-x
toxicity_safety_v0_1,train,toxicity_endpoint,522,toxicity,A,"iExoKrasG12D (recovered B2 from PMID:41027940, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Engineered exosomes with Kras(G12D) specific siRNA in pancreatic cancer: a phase I study with immunological correlates.,source:1803|molecule:1013,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41027940,10.1038/s41467-025-63718-2
toxicity_safety_v0_1,train,toxicity_endpoint,527,toxicity,B,"STP705 (recovered B2 from PMID:39692702, curator:CM)",siRNA,,,hepatic,hepatotoxicity,A Novel Injectable Polypeptide Nanoparticle Encapsulated siRNA Targeting TGF-β1 and COX-2 for Localized Fat Reduction II: Phase I Clinical Trial.,source:2535|molecule:1067,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39692702,10.1111/jocd.16722
toxicity_safety_v0_1,train,toxicity_endpoint,530,toxicity,A,siRNAs,siRNA,,,hepatic,hepatotoxicity,Potent and durable gene modulation in heart and muscle with chemically defined lipophilic siRNAs.,source:3407|molecule:435,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41432033,10.1093/nar/gkaf1374
toxicity_safety_v0_1,train,toxicity_endpoint,531,toxicity,A,elebsiran,siRNA,,,hepatic,hepatotoxicity,An RNA interference therapeutic potentially achieves functional cure of chronic hepatitis B virus infection.,source:3426|molecule:525,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41326382,10.1038/s41467-025-66876-5
toxicity_safety_v0_1,train,toxicity_endpoint,538,toxicity,B,unspecified ASO (PMID:40736236),ASO,,,general safety,cell viability/cytotoxicity,An antisense peptide-conjugated peptide nucleic acid (PPNA) for peptidoglycan recycling inhibition reduces AmpC hyperproduction and β-lactam resistance in Pseudomonas aeruginosa.,source:3573|molecule:1068,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40736236,10.1128/spectrum.02622-24
toxicity_safety_v0_1,train,toxicity_endpoint,543,toxicity,A,"HSA@ICG/siRNA NPs (recovered B2 from PMID:40259913, curator:CM)",siRNA,,,hepatic,hepatotoxicity,NIR-Triggered siRNA Release and Lysosomal Escape for Synergistic Photothermal Tumor Therapy.,source:3710|molecule:1017,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40259913,10.2147/IJN.S511655
toxicity_safety_v0_1,train,toxicity_endpoint,544,toxicity,A,unspecified siRNA (PMID:40143003),siRNA,,,general safety,cell viability/cytotoxicity,Analysis of Polymer/siRNA Nanoparticle Efficacy and Biocompatibility in 3D Air-Liquid Interface Culture Compared to 2D Cell Culture.,source:3725|molecule:1018,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40143003,10.3390/pharmaceutics17030339
toxicity_safety_v0_1,train,toxicity_endpoint,546,toxicity,A,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,renal,renal safety,A Novel Approach for In Vitro Testing and Hazard Evaluation of Nanoformulated RyR2-Targeting siRNA Drugs Using Human PBMCs.,source:3771|molecule:985,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39860035,10.3390/life15010095
toxicity_safety_v0_1,train,toxicity_endpoint,560,toxicity,A,unspecified siRNA (PMID:41226635),siRNA,,,hepatic,hepatotoxicity,Anti-Angiogenic RNAi-Based Treatment of Endometriosis in a Rat Model Using CXCR4-Targeted Peptide Nanoparticles.,source:7835|molecule:1020,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41226635,10.3390/ijms262110582
toxicity_safety_v0_1,train,toxicity_endpoint,566,toxicity,A,siPD-L1,siRNA,,,hepatic,hepatotoxicity,Modulating tumor collagen fiber alignment for enhanced lung cancer immunotherapy via inhaled RNA.,source:8508|molecule:531,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40885724,10.1038/s41467-025-63415-0
toxicity_safety_v0_1,train,toxicity_endpoint,568,toxicity,B,unspecified siRNA (PMID:40848064),siRNA,,,general safety,cell viability/cytotoxicity,"Heat up, silence on: IDO1 gene silencing in THP-1-derived dendritic cells triggered by magnetic hyperthermia.",source:8603|molecule:1070,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40848064,10.1007/s00262-025-04148-3
toxicity_safety_v0_1,train,toxicity_endpoint,574,toxicity,B,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic,hepatotoxicity,Intravenous Administration of sRNA Nanoparticles for Treatment of Osteoporosis in Mice.,source:9180|molecule:986,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40574101,10.3390/pharmaceutics17060789
toxicity_safety_v0_1,train,toxicity_endpoint,581,toxicity,A,unspecified siRNA (PMID:40297405),siRNA,,,hepatic,hepatotoxicity,ROS-Responsive Biomimetic Nanocomplexes of Liposomes and Macrophage-Derived Exosomes for Combination Breast Cancer Therapy.,source:9662|molecule:1022,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40297405,10.2147/IJN.S514375
toxicity_safety_v0_1,train,toxicity_endpoint,586,toxicity,B,unspecified siRNA (PMID:40207309),siRNA,,,general toxicity,toxicity,Si-ITGA6-Loaded Liposomes Inhibit Capsule Fibrosis via the FAK/PI3K/Akt Signaling Pathway in Adhesive Capsulitis of Shoulder.,source:9792|molecule:1071,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40207309,10.2147/IJN.S501561
toxicity_safety_v0_1,train,toxicity_endpoint,587,toxicity,A,nusinersen,ASO,,,renal,renal safety,Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2S.,source:9966|molecule:299,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40060931,10.1016/j.omtn.2025.102479
toxicity_safety_v0_1,train,toxicity_endpoint,589,toxicity,A,unspecified CpG (PMID:39838063),ASO,,,hepatic,hepatotoxicity,Preclinical assessment of splicing modulation therapy for ABCA4 variant c.768G>T in Stargardt disease.,source:10245|molecule:1023,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39838063,10.1038/s43856-024-00712-7
toxicity_safety_v0_1,train,toxicity_endpoint,600,toxicity,A,"ION-827359 (recovered B2 from PMID:39286058, curator:CM)",ASO,,,hepatic,hepatotoxicity,"Randomised, phase 1/2a trial of ION-827359, an antisense oligonucleotide inhibitor of ENaC.",source:1562|molecule:1025,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39286058,10.1183/23120541.00986-2023
toxicity_safety_v0_1,train,toxicity_endpoint,602,toxicity,A,siRNA-mediated,siRNA,,,hepatic,hepatotoxicity,siRNA-mediated reduction of a circulating protein in swine using lipid nanoparticles.,source:2588|molecule:472,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38779336,10.1016/j.omtm.2024.101258
toxicity_safety_v0_1,train,toxicity_endpoint,603,toxicity,B,unspecified ASO (PMID:38276948),ASO,,,hepatic,hepatotoxicity,An antisense oligonucleotide efficiently suppresses splicing of an alternative exon in vascular smooth muscle in vivo.,source:2618|molecule:1072,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38276948,10.1152/ajpheart.00745.2023
toxicity_safety_v0_1,train,toxicity_endpoint,605,toxicity,B,unspecified siRNA (PMID:39296267),siRNA,,,hepatic,hepatotoxicity,Lipopolymer/siRNA Nanoparticles Targeting the Signal Transducer and Activator of Transcription 5A Disrupts Proliferation of Acute Lymphoblastic Leukemia.,source:3848|molecule:1073,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39296267,10.1021/acsptsci.4c00336
toxicity_safety_v0_1,train,toxicity_endpoint,608,toxicity,A,unspecified siRNA (PMID:38617800),siRNA,,,general safety,cell viability/cytotoxicity,Microdroplets Encapsulated with NFATc1-siRNA and Exosomes-Derived from MSCs Onto 3D Porous PLA Scaffold for Regulating Osteoclastogenesis and Promoting Osteogenesis.,source:3912|molecule:1027,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38617800,10.2147/IJN.S443413
toxicity_safety_v0_1,train,toxicity_endpoint,609,toxicity,B,unspecified siRNA (PMID:38613388),ASO,,,renal,renal safety,Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo.,source:3913|molecule:1074,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38613388,10.1093/nar/gkae260
toxicity_safety_v0_1,train,toxicity_endpoint,623,toxicity,A,fitusiran,siRNA,,,hepatic,hepatotoxicity,Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial.,source:11416|molecule:551,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38452197,10.1182/blood.2023021864
toxicity_safety_v0_1,train,toxicity_endpoint,628,toxicity,A,"cRGD-GR9G-(LLHH)3/siEGFR/siP65 (recovered B2 from PMID:39539970, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Stimuli-Responsive Peptide/siRNA Nanoparticles as a Radiation Sensitizer for Glioblastoma Treatment by Co-Inhibiting RELA/P65 and EGFR.,source:12446|molecule:1028,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39539970,10.2147/IJN.S483252
toxicity_safety_v0_1,train,toxicity_endpoint,639,toxicity,A,unspecified ASO (PMID:38042877),ASO,,,hepatic,hepatotoxicity,Dynamic and static control of the off-target interactions of antisense oligonucleotides using toehold chemistry.,source:1136|molecule:1029,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38042877,10.1038/s41467-023-43714-0
toxicity_safety_v0_1,train,toxicity_endpoint,640,toxicity,A,unspecified siRNA,siRNA,,,general safety,cell viability/cytotoxicity,Antiviral Efficacy of RNase H-Dependent Gapmer Antisense Oligonucleotides against Japanese Encephalitis Virus.,source:1292|molecule:185,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,37834294,10.3390/ijms241914846
toxicity_safety_v0_1,train,toxicity_endpoint,641,toxicity,B,unspecified ASO,ASO,,,hepatic,hepatotoxicity,"The exon-skipping oligonucleotide, KitStop, depletes tissue-resident mast cells in vivo to ameliorate anaphylaxis.",source:1955|molecule:494,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,36798116,10.3389/fimmu.2023.1006741
toxicity_safety_v0_1,train,toxicity_endpoint,646,toxicity,A,"9b2KC-23b (recovered B2 from PMID:37744174, curator:CM)",PMO,,,hepatic,hepatotoxicity,Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression.,source:11931|molecule:1032,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,37744174,10.1016/j.omtn.2023.09.001
toxicity_safety_v0_1,train,toxicity_endpoint,648,toxicity,A,unspecified ASO (PMID:37704069),ASO,,,hepatic,hepatotoxicity,Antisense oligonucleotides targeting a NOTCH3 mutation in male mice ameliorate the cortical osteopenia of lateral meningocele syndrome.,source:12175|molecule:1033,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,37704069,10.1016/j.bone.2023.116898
toxicity_safety_v0_1,train,toxicity_endpoint,654,toxicity,A,"RO7062931 (recovered B2 from PMID:37147890, curator:CM)",ASO,,,hepatic,hepatotoxicity,A single ascending dose study of single-stranded oligodeoxyribonucleotide RO7062931 in Chinese healthy volunteers.,source:14152|molecule:1035,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,37147890,10.1111/cts.13531
toxicity_safety_v0_1,train,toxicity_endpoint,657,toxicity,B,single-ascending,ASO,,,hepatic,hepatotoxicity,"A multi-purpose Japanese phase I study in the global development of vupanorsen: Randomized, placebo-controlled, single-ascending dose study in adults.",source:14202|molecule:559,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,37002654,10.1111/cts.13498
toxicity_safety_v0_1,train,toxicity_endpoint,659,toxicity,A,unspecified siRNA (PMID:36614194),siRNA,,,hepatic,hepatotoxicity,Target-Specific Nanoparticle Polyplex Down-Regulates Mutant Kras to Prevent Pancreatic Carcinogenesis and Halt Tumor Progression.,source:14302|molecule:1037,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,36614194,10.3390/ijms24010752
toxicity_safety_v0_1,train,toxicity_endpoint,660,toxicity,A,unspecified ASO (PMID:35861704),ASO,,,hepatic,hepatotoxicity,The Combination of Mesyl-Phosphoramidate Inter-Nucleotide Linkages and 2'-O-Methyl in Selected Positions in the Antisense Oligonucleotide Enhances the Performance of RNaseH1 Active PS-ASOs.,source:1485|molecule:1038,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,35861704,10.1089/nat.2022.0005
toxicity_safety_v0_1,train,toxicity_endpoint,661,toxicity,A,"ASO-001933 (recovered B2 from PMID:36090761, curator:CM)",ASO,,,hepatic,hepatotoxicity,Identification and characterization of a MAPT-targeting locked nucleic acid antisense oligonucleotide therapeutic for tauopathies.,source:1615|molecule:1039,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,36090761,10.1016/j.omtn.2022.07.027
toxicity_safety_v0_1,train,toxicity_endpoint,662,toxicity,A,single-dose,ASO,,,hepatic,hepatotoxicity,"Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models.",source:1617|molecule:561,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,35860385,10.1016/j.omtn.2022.06.015
toxicity_safety_v0_1,train,toxicity_endpoint,666,toxicity,A,"IONIS-FXIRx (recovered B2 from PMID:35155859, curator:CM)",ASO,,,hepatic,hepatotoxicity,Phase 2 Study of the Factor XI Antisense Inhibitor IONIS-FXI(Rx) in Patients With ESRD.,source:1981|molecule:1041,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,35155859,10.1016/j.ekir.2021.11.011
toxicity_safety_v0_1,train,toxicity_endpoint,668,toxicity,A,"ASO 5–2 (recovered B2 from PMID:34949835, curator:CM)",ASO,,,hepatic,hepatotoxicity,Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.,source:2727|molecule:1042,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,34949835,10.1038/s41591-021-01557-6
toxicity_safety_v0_1,train,toxicity_endpoint,676,toxicity,A,unspecified ASO (PMID:34921016),ASO,,,hepatic,hepatotoxicity,ASO-Based PKM Splice-Switching Therapy Inhibits Hepatocellular Carcinoma Growth.,source:14660|molecule:1044,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,34921016,10.1158/0008-5472.CAN-20-0948
toxicity_safety_v0_1,train,toxicity_endpoint,678,toxicity,B,Site-specific,ASO,,,hepatic,hepatotoxicity,"Site-specific Incorporation of 2',5'-Linked Nucleic Acids Enhances Therapeutic Profile of Antisense Oligonucleotides.",source:1638|molecule:563,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,34141070,10.1021/acsmedchemlett.1c00072
toxicity_safety_v0_1,train,toxicity_endpoint,687,toxicity,B,"MRCV-19 (recovered B2 from PMID:34440222, curator:CM)",PMO,,,hepatic,hepatotoxicity,Vivo-Morpholino-Based Antiviral for SARS-CoV-2: Implications for Novel Therapies in the Treatment of Acute COVID-19 Disease.,source:14753|molecule:1076,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,34440222,10.3390/biomedicines9081018
toxicity_safety_v0_1,train,toxicity_endpoint,695,toxicity,A,"NVP-13 (recovered B2 from PMID:32178467, curator:CM)",ASO,,,hepatic,hepatotoxicity,Antisense Oligonucleotide in LNA-Gapmer Design Targeting TGFBR2-A Key Single Gene Target for Safe and Effective Inhibition of TGFβ Signaling.,source:1492|molecule:1047,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,32178467,10.3390/ijms21061952
toxicity_safety_v0_1,train,toxicity_endpoint,696,toxicity,A,"LNAc/2′OMe18h (recovered B2 from PMID:31873063, curator:CM)",ASO,,,hepatic,hepatotoxicity,Systemic Evaluation of Chimeric LNA/2'-O-Methyl Steric Blockers for Myotonic Dystrophy Type 1 Therapy.,source:1660|molecule:1048,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,31873063,10.1089/nat.2019.0811
toxicity_safety_v0_1,train,toxicity_endpoint,700,toxicity,A,unspecified ASO (PMID:32805488),ASO,,,hepatic,hepatotoxicity,MYC ASO Impedes Tumorigenesis and Elicits Oncogene Addiction in Autochthonous Transgenic Mouse Models of HCC and RCC.,source:14986|molecule:1049,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,32805488,10.1016/j.omtn.2020.07.008
toxicity_safety_v0_1,train,toxicity_endpoint,702,toxicity,A,unspecified ASO (PMID:30988454),ASO,,,hepatic,hepatotoxicity,Systematic evaluation of 2'-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping in vitro.,source:2782|molecule:1050,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,30988454,10.1038/s41598-019-42523-0
toxicity_safety_v0_1,train,toxicity_endpoint,703,toxicity,A,Site-specific,ASO,,,hepatic,hepatotoxicity,Site-specific replacement of phosphorothioate with alkyl phosphonate linkages enhances the therapeutic profile of gapmer ASOs by modulating interactions with cellular proteins.,source:3148|molecule:563,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,31034558,10.1093/nar/gkz247
toxicity_safety_v0_1,train,toxicity_endpoint,705,toxicity,A,unspecified siRNA (PMID:31375812),siRNA,,,hepatic,hepatotoxicity,A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.,source:4213|molecule:990,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,31375812,10.1038/s41587-019-0205-0
toxicity_safety_v0_1,train,toxicity_endpoint,706,toxicity,A,"IONIS-PKKRx (recovered B2 from PMID:30817230, curator:CM)",ASO,,,hepatic,hepatotoxicity,IONIS-PKK(Rx) a Novel Antisense Inhibitor of Prekallikrein and Bradykinin Production.,source:12610|molecule:1051,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,30817230,10.1089/nat.2018.0754
toxicity_safety_v0_1,train,toxicity_endpoint,708,toxicity,A,"BMSP-PMO-GF (recovered B2 from PMID:31629961, curator:CM)",PMO,,,hepatic,hepatotoxicity,Hexose Potentiates Peptide-Conjugated Morpholino Oligomer Efficacy in Cardiac Muscles of Dystrophic Mice in an Age-Dependent Manner.,source:15084|molecule:1052,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,31629961,10.1016/j.omtn.2019.09.012
toxicity_safety_v0_1,train,toxicity_endpoint,723,toxicity,A,silk,CpG oligodeoxynucleotide,,,general safety,cell viability/cytotoxicity,Functionalized bioengineered spider silk spheres improve nuclease resistance and activity of oligonucleotide therapeutics providing a strategy for cancer treatment.,source:12648|molecule:575,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,28694238,10.1016/j.actbio.2017.07.014
toxicity_safety_v0_1,train,toxicity_endpoint,729,toxicity,A,unspecified siRNA (PMID:27483025),siRNA,,,hepatic,hepatotoxicity,Silencing Myostatin Using Cholesterol-conjugated siRNAs Induces Muscle Growth.,source:12672|molecule:1054,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,27483025,10.1038/mtna.2016.55
toxicity_safety_v0_1,train,toxicity_endpoint,731,toxicity,A,DUX4,PMO,,,hepatic,hepatotoxicity,Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics.,source:15206|molecule:578,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,27378237,10.1038/mt.2016.111
toxicity_safety_v0_1,train,toxicity_endpoint,736,toxicity,B,unspecified ASO,ASO,,,general toxicity,toxicity,In vitro and in vivo activity of a novel locked nucleic acid (LNA)-inhibitor-miR-221 against multiple myeloma cells.,source:4256|molecule:494,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,24586944,10.1371/journal.pone.0089659
toxicity_safety_v0_1,train,toxicity_endpoint,742,toxicity,B,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic,hepatotoxicity,An exocyclic methylene group acts as a bioisostere of the 2'-oxygen atom in LNA.,source:4288|molecule:986,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,20886816,10.1021/ja105875e
toxicity_safety_v0_1,train,toxicity_endpoint,958,toxicity,B,SQY51,ASO,,,general safety,cell viability/cytotoxicity,Systemic distribution of tricyclo-DNA antisense oligonucleotide following intratracheal instillation in the mouse.,source:1213|molecule:633,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41583579,10.1093/narmme/ugag001
toxicity_safety_v0_1,train,toxicity_endpoint,966,toxicity,A,"siRMSTN (recovered B2 from PMID:41041909, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Silencing Myostatin Using In Vivo Self-Assembled siRNA Protects Against Cancer- and Dexamethasone-Induced Muscle Atrophy.,source:1776|molecule:1055,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41041909,10.1002/adhm.202502186
toxicity_safety_v0_1,train,toxicity_endpoint,969,toxicity,A,"APGS (recovered B2 from PMID:41732207, curator:CM)",ASO,,,hepatic,hepatotoxicity,"Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer's disease.",source:3251|molecule:1056,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41732207,10.1016/j.omtn.2026.102848
toxicity_safety_v0_1,train,toxicity_endpoint,974,toxicity,A,"S9 (recovered B2 from PMID:41552385, curator:CM)",siRNA,,,hepatic,hepatotoxicity,An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment.,source:3301|molecule:994,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41552385,10.1016/j.omtn.2025.102802
toxicity_safety_v0_1,train,toxicity_endpoint,1039,toxicity,A,unspecified siRNA (PMID:40737088),siRNA,,,hepatic,hepatotoxicity,Single alkyl phosphonate modification of the siRNA backbone in the seed region enhances specificity and therapeutic profile.,source:1497|molecule:1057,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40737088,10.1093/nar/gkaf692
toxicity_safety_v0_1,train,toxicity_endpoint,1057,toxicity,A,Single-dose,siRNA,,,general toxicity,toxicity,Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model.,source:3701|molecule:652,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40313754,10.21203/rs.3.rs-6465542/v1
toxicity_safety_v0_1,train,toxicity_endpoint,1083,toxicity,A,BHF7,siRNA,,,hepatic,hepatotoxicity,Small Interfering RNA Therapy Targeting the Long Noncoding RNA SMILR for Therapeutic Intervention in Coronary Artery Bypass Graft Failure.,source:8415|molecule:660,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40928446,10.1016/j.jacbts.2025.101364
toxicity_safety_v0_1,train,toxicity_endpoint,1117,toxicity,A,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic,hepatotoxicity,Therapeutic Potential of STE20-Type Kinase STK25 Inhibition for the Prevention and Treatment of Metabolically Induced Hepatocellular Carcinoma.,source:10025|molecule:986,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40024534,10.1016/j.jcmgh.2025.101485
toxicity_safety_v0_1,train,toxicity_endpoint,1141,toxicity,A,"siTMEM45A (recovered B2 from PMID:39910045, curator:CM)",siRNA,,,hepatic,hepatotoxicity,TMEM45A enhances palbociclib resistance and cellular glycolysis by activating AKT/mTOR signaling pathway in HR+ breast cancer.,source:13436|molecule:1058,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39910045,10.1038/s41420-025-02336-9
toxicity_safety_v0_1,train,toxicity_endpoint,1161,toxicity,A,givosiran,siRNA,,,hepatic,hepatotoxicity,German Real-World Experience of Patients with Diverse Features of Acute Intermittent Porphyria Treated with Givosiran.,source:10566|molecule:231,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39597922,10.3390/jcm13226779
toxicity_safety_v0_1,train,toxicity_endpoint,1172,toxicity,A,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic,hepatotoxicity,RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice.,source:10988|molecule:985,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38979291,10.1101/2024.06.20.599943
toxicity_safety_v0_1,train,toxicity_endpoint,1179,toxicity,B,"OA2-siRAPSYN (recovered B2 from PMID:38741123, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Targeted degradation of oncogenic BCR-ABL by silencing the gene of NEDD8 E3 ligase RAPSYN.,source:11191|molecule:1078,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38741123,10.1186/s12951-024-02505-5
toxicity_safety_v0_1,train,toxicity_endpoint,1180,toxicity,B,nusinersen,ASO,,,general safety,systemic safety/tolerability,A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results.,source:11199|molecule:299,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38722537,10.1007/s12325-024-02852-7
toxicity_safety_v0_1,train,toxicity_endpoint,1203,toxicity,A,unspecified ASO (PMID:37200862),ASO,,,hepatic,hepatotoxicity,Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1.,source:1481|molecule:1060,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,37200862,10.1016/j.omtn.2023.04.010
toxicity_safety_v0_1,train,toxicity_endpoint,1214,toxicity,A,"si3033 (recovered B2 from PMID:37925520, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin.,source:3979|molecule:1061,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,37925520,10.1038/s41467-023-42714-4
toxicity_safety_v0_1,train,toxicity_endpoint,1240,toxicity,B,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic,hepatotoxicity,ANGPTL4 silencing via antisense oligonucleotides reduces plasma triglycerides and glucose in mice without causing lymphadenopathy.,source:1619|molecule:986,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,35667416,10.1016/j.jlr.2022.100237
toxicity_safety_v0_1,train,toxicity_endpoint,1244,toxicity,A,"SMN7D(-10-29) AO (recovered B2 from PMID:35464859, curator:CM)",ASO,,,hepatic,hepatotoxicity,"Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In Vitro.",source:2210|molecule:1062,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,35464859,10.3389/fgene.2022.791416
toxicity_safety_v0_1,train,toxicity_endpoint,1257,toxicity,A,inotersen,ASO,,,hematological,thrombocytopenia,Real-life experience with inotersen in hereditary transthyretin amyloidosis with late-onset phenotype: Data from an early-access program in Italy.,source:14599|molecule:599,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,35289020,10.1111/ene.15325
toxicity_safety_v0_1,train,toxicity_endpoint,1263,toxicity,A,Single-Stranded,ASO,,,hepatic,hepatotoxicity,Clinical Study of Single-Stranded Oligonucleotide RO7062931 in Healthy Volunteers and Patients With Chronic Hepatitis B.,source:1996|molecule:695,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,34037271,10.1002/hep.31920
toxicity_safety_v0_1,train,toxicity_endpoint,1270,toxicity,A,siRNAs,siRNA,,,hepatic,hepatotoxicity,A smart multiantenna gene theranostic system based on the programmed assembly of hypoxia-related siRNAs.,source:12553|molecule:435,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,34172725,10.1038/s41467-021-24191-9
toxicity_safety_v0_1,train,toxicity_endpoint,1293,toxicity,A,siRNAs,siRNA,,,hepatic,hepatotoxicity,Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways.,source:12616|molecule:435,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,30535404,10.1093/nar/gky1232
toxicity_safety_v0_1,train,toxicity_endpoint,1306,toxicity,B,"MORF1-MORF2 (recovered B2 from PMID:24308267, curator:CM)",PMO,,,hepatic,hepatotoxicity,Cell surface self-assembly of hybrid nanoconjugates via oligonucleotide hybridization induces apoptosis.,source:4261|molecule:1080,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,24308267,10.1021/nn4053827
toxicity_safety_v0_1,validation,toxicity_endpoint,187,toxicity,A,drisapersen,ASO/siRNA mixed context,,,hepatic,hepatotoxicity,Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial.,source:24321374|molecule:drisapersen,source_plus_molecule_grouped_manual_v1,20260602_batch003_manual_benchmark_v1,24321374,10.1016/j.nmd.2013.09.004
toxicity_safety_v0_1,validation,toxicity_endpoint,426,toxicity,A,siRNA nanocarriers/lipoplexes,siRNA,,,general safety,cell viability/cytotoxicity,Pulmonary delivery of siRNA lipoplexes and lipid nanoparticles using a vibrating mesh nebuliser.,source:41274361|molecule:siRNA nanocarriers/lipoplexes,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41274361,10.1016/j.ejps.2025.107386
toxicity_safety_v0_1,validation,toxicity_endpoint,432,toxicity,A,siRNA-loaded CaP-NP/cGM formulation,siRNA,Chordin,,general safety,cell viability/biocompatibility,A smart nanocomposite bioactive ink for controlled siRNA delivery in calvarial mesenchymal stromal cells as a minimally invasive treatment for craniosynostosis.,source:41647289|molecule:siRNA-loaded CaP-NP/cGM formulation,source_plus_molecule_grouped_manual_v1,20260602_batch004_007_manual_benchmark_v1,41647289,10.1093/rb/rbaf115
toxicity_safety_v0_1,validation,toxicity_endpoint,452,toxicity,A,siRNA SORT LNPs,siRNA,,,general safety,systemic safety/tolerability,"Expanding RNAi to Kidneys, Lungs, and Spleen via Selective ORgan Targeting (SORT) siRNA Lipid Nanoparticles.",source:2577|molecule:408,source_plus_molecule_grouped_hash_v1,release_scale_review_batch008_manual_benchmark_v1,38973655,10.1002/adma.202313791
toxicity_safety_v0_1,validation,toxicity_endpoint,477,toxicity,A,"TLK2 ASO (recovered B2 from PMID:41878536, curator:CM)",ASO,,,hepatic,hepatotoxicity,Targeting TLK2 with antisense oligonucleotides as a new strategy in acute myeloid leukemia.,source:1062|molecule:1006,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41878536,10.3389/fonc.2026.1659341
toxicity_safety_v0_1,validation,toxicity_endpoint,479,toxicity,A,siRNA-Lipid,siRNA,,,hepatic,hepatotoxicity,DNA Repair Enzyme Regulation Strategy for Enhanced Pancreatic Neuroendocrine Tumor Therapy via Targeting siRNA-Lipid Nanoparticles.,source:1193|molecule:507,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41919978,10.1021/acsnano.5c21452
toxicity_safety_v0_1,validation,toxicity_endpoint,533,toxicity,A,"KFFK (FFK)2-conjugated PNA (recovered B2 from PMID:41096791, curator:CM)",ASO,,,general safety,cell viability/cytotoxicity,Targeting carA Using Optimized Antisense Peptide Nucleic Acid-Cell-Penetrating Peptide Conjugates in Acinetobacter baumannii: A Novel Antibacterial Approach.,source:3472|molecule:1015,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41096791,10.3390/ijms26199526
toxicity_safety_v0_1,validation,toxicity_endpoint,548,toxicity,A,siRNAs,siRNA,,,hepatic,hepatotoxicity,"Design, Screening and Development of Asymmetric siRNAs Targeting the MYC Oncogene in Triple-Negative Breast Cancer.",source:3801|molecule:435,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39632755,10.4062/biomolther.2024.071
toxicity_safety_v0_1,validation,toxicity_endpoint,556,toxicity,A,"siPCSK9 (recovered B2 from PMID:41290746, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Silencing PCSK9 reshapes the spatiotemporal activation of STING for safe and effective cancer immunotherapy.,source:7717|molecule:1019,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,41290746,10.1038/s41467-025-66630-x
toxicity_safety_v0_1,validation,toxicity_endpoint,565,toxicity,B,"PUMCH-E13 (recovered B2 from PMID:40970667, curator:CM)",ASO,,,hepatic,hepatotoxicity,Exon Skipping Therapy Restores Ciliary Function in USH2A-Related Retinal Degeneration.,source:8330|molecule:1069,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40970667,10.1167/iovs.66.12.46
toxicity_safety_v0_1,validation,toxicity_endpoint,598,toxicity,A,unspecified ASO (PMID:39460376),ASO,,,hepatic,hepatotoxicity,Quantifying and mitigating motor phenotypes induced by antisense oligonucleotides in the central nervous system.,source:1282|molecule:1024,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39460376,10.1016/j.ymthe.2024.10.024
toxicity_safety_v0_1,validation,toxicity_endpoint,642,toxicity,A,unspecified siRNA (PMID:36727438),ASO,,,hepatic,hepatotoxicity,Simultaneous inhibition of endocytic recycling and lysosomal fusion sensitizes cells and tissues to oligonucleotide therapeutics.,source:1958|molecule:1030,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,36727438,10.1093/nar/gkad023
toxicity_safety_v0_1,validation,toxicity_endpoint,644,toxicity,A,unspecified ASO (PMID:38015828),ASO,,,hepatic,hepatotoxicity,Targeted ASO-mediated Atp1a2 knockdown in astrocytes reduces SOD1 aggregation and accelerates disease onset in mutant SOD1 mice.,source:11827|molecule:1031,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38015828,10.1371/journal.pone.0294731
toxicity_safety_v0_1,validation,toxicity_endpoint,650,toxicity,A,"iLANDsiPAK4 (recovered B2 from PMID:37719367, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Antitumor synergism between PAK4 silencing and immunogenic phototherapy of engineered extracellular vesicles.,source:13969|molecule:1034,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,37719367,10.1016/j.apsb.2023.03.020
toxicity_safety_v0_1,validation,toxicity_endpoint,651,toxicity,B,single-chain,ASO,,,renal,renal safety,Preventive treatment of coronavirus disease-2019 virus using coronavirus disease-2019-receptor-binding domain 1C aptamer by suppress the expression of angiotensin-converting enzyme 2 receptor.,source:13975|molecule:557,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,37692001,10.4103/JAPTR.JAPTR_117_23
toxicity_safety_v0_1,validation,toxicity_endpoint,658,toxicity,A,"DG9-PMO (recovered B2 from PMID:36719755, curator:CM)",PMO,,,immunotoxicity,immune activation,DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration.,source:14276|molecule:1036,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,36719755,10.1172/jci.insight.160516
toxicity_safety_v0_1,validation,toxicity_endpoint,686,toxicity,A,"CpG2018B (recovered B2 from PMID:34566407, curator:CM)",CpG oligodeoxynucleotide,,,hepatic,hepatotoxicity,CpG Oligodeoxynucleotide Developed to Activate Primate Immune Responses Promotes Antitumoral Effects in Combination with a Neoantigen-Based mRNA Cancer Vaccine.,source:14741|molecule:1045,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,34566407,10.2147/DDDT.S325790
toxicity_safety_v0_1,validation,toxicity_endpoint,690,toxicity,B,sided,ASO,,,general toxicity,toxicity,Phase 3 randomized trial of chemotherapy with or without oblimersen in older AML patients: CALGB 10201 (Alliance).,source:14789|molecule:569,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,34251414,10.1182/bloodadvances.2021004233
toxicity_safety_v0_1,validation,toxicity_endpoint,1043,toxicity,A,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hematological,thrombocytopenia,Rats subject to extracorporeal membrane oxygenation have improved cardiac function following anticoagulation and reversal with factor IXa aptamer-antidote oligonucleotide pair.,source:1828|molecule:986,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,40599554,10.1097/CP9.0000000000000122
toxicity_safety_v0_1,validation,toxicity_endpoint,1131,toxicity,A,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,renal,renal safety,"Cetuximab-Immunoliposomes Loaded with TGF-β1 siRNA for the Targeting Therapy of NSCLC: Design, and In Vitro and In Vivo Evaluation.",source:12392|molecule:985,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,39940962,10.3390/ijms26031196
toxicity_safety_v0_1,validation,toxicity_endpoint,1156,toxicity,A,unspecified siRNA (PMID:38192611),siRNA,,,hepatic,hepatotoxicity,Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors.,source:3958|molecule:1059,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,38192611,10.1016/j.omtn.2023.102088
toxicity_safety_v0_1,validation,toxicity_endpoint,1247,toxicity,A,"PC-DCA siRNA (recovered B2 from PMID:35847173, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1.,source:4069|molecule:1063,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,35847173,10.1016/j.omtn.2022.06.009
toxicity_safety_v0_1,validation,toxicity_endpoint,1292,toxicity,B,"RGD-PEG-ECO/siβ3 (recovered B2 from PMID:31140918, curator:CM)",siRNA,,,hepatic,hepatotoxicity,Formulation of Biocompatible Targeted ECO/siRNA Nanoparticles with Long-Term Stability for Clinical Translation of RNAi.,source:12604|molecule:1079,source_plus_molecule_grouped_hash_v1,release_scale_review_batch009_mega_fast_manual_benchmark_v1,31140918,10.1089/nat.2019.0784