Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DU7XQ3
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| Drug Name |
BIIB112
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| Synonyms |
cotoretigene toliparvovec
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| Drug Type |
Gene therapy
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| Indication | X-linked retinitis pigmentosa [ICD-11: 9B70] | Phase 3 | [1] | |
| Company |
Biogen
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | X-linked retinitis pigmentosa GTPase regulator (RPGR) | Target Info | . | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03584165) A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects With Choroideremia Previously Treated With Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects With X-Linked Retinitis Pigmentosa Previously Treated With Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study. U.S.National Institutes of Health. | |||
| REF 2 | Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations. JAMA Ophthalmol. 2023 Mar 1;141(3):275-283. | |||
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