Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DM2J3CX)

Drug Name

ION363 Drug Info

Indication

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

Cross-matching ID

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Download the Complete Interaction Atlas for Visualization in Cytoscape

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]

------------------------------------------------------------------------------------

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.