General Information of Drug (ID: DM2J3CX)

Drug Name
ION363
Indication
Disease Entry ICD 11 Status REF
Amyotrophic lateral sclerosis 8B60.0 Phase 3 [1]
Drug Type
Antisense oligonucleotide
Cross-matching ID
TTD ID
DCRJ78

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
RNA-binding protein FUS messenger RNA (FUS mRNA) TTKGYZ9 FUS_HUMAN Not Available [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

1 ClinicalTrials.gov (NCT04768972) A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients With Fused in Sarcoma Mutations (FUS-ALS). U.S.National Institutes of Health.
2 Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis. Nat Med. 2022 Jan;28(1):104-116.