Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DCRJ78
|
|||
| Drug Name |
ION363
|
|||
| Drug Type |
Antisense oligonucleotide
|
|||
| Indication | Amyotrophic lateral sclerosis [ICD-11: 8B60.0; ICD-9: 335.2] | Phase 3 | [1] | |
| Company |
Ionis
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | RNA-binding protein FUS messenger RNA (FUS mRNA) | Target Info | . | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 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. | |||
| REF 2 | Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis. Nat Med. 2022 Jan;28(1):104-116. | |||
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