Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DOE96M
|
|||
| Drug Name |
Belcesiran
|
|||
| Drug Type |
RNA interference
|
|||
| Indication | Alpha-1 antitrypsin deficiency [ICD-11: 5C5A; ICD-10: E88.0, M79.3; ICD-9: 273.4] | Phase 2 | [1] | |
| Company |
Dicerna Pharmaceuticals
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | SERPINA1 messenger RNA (SERPINA1 mRNA) | Target Info | . | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04764448) A Phase 2, Randomized, Double-blind, Placebo-Controlled Study Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two Dose Levels of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency-Associated Liver Disease. U.S.National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Dicerna Pharmaceuticals | |||
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