Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DS14FO
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| Drug Name |
Sepofarsen
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| Synonyms |
QR-110
Click to Show/Hide
|
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| Drug Type |
Oligonucleotide
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| Indication | Leber congenital amaurosis [ICD-11: 9B70; ICD-10: H35.5] | Phase 2/3 | [1] | |
| Company |
ProQR Therapeutics
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | CEP290 messenger RNA (CEP290 mRNA) | Target Info | Inhibitor | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04855045) An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. (BRIGHTEN). U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of ProQR Therapeutics. | |||
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