Details of the Drug

General Information of Drug (ID: DM2J3CX)

Drug Name

ION363

Indication

Disease Entry	ICD 11	Status	REF
Amyotrophic lateral sclerosis	8B60.0	Phase 3	[1]
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Drug Type

Antisense oligonucleotide

Cross-matching ID

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]

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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.