Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DUWB31
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| Drug Name |
BIIB115
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| Synonyms |
ION306
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| Drug Type |
Antisense oligonucleotide
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| Indication | Spinal muscular atrophy [ICD-11: 8B61] | Phase 1 | [1] | |
| Company |
Ionis
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | SMN1 messenger RNA (SMN1 mRNA) | Target Info | . | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT05575011) A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previously Treated With Onasemnogene Abeparvovec (Zolgensma?) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115. U.S.National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Ionis | |||
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