Drug Profile
Botaretigene sparoparvovec - Johnson & Johnson Innovative Medicine/MeiraGTx
Alternative Names: A-004; AAV-RPGR - MeiraGTx; AAV2/5-hRKp.RPGR - MeiraGTx; AAV2/5-RPGR; AAV5-hRKp.RPGR; AAV5-RPGR; RPGR AAV gene therapyLatest Information Update: 10 Oct 2023
Price :
$50
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At a glance
- Originator MeiraGTx
- Developer Johnson & Johnson Innovative Medicine; MeiraGTx
- Class Eye disorder therapies; Gene therapies
- Mechanism of Action Gene transference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Phase III Retinitis pigmentosa
Most Recent Events
- 01 Oct 2023 Janssen Pharmaceuticals is now called Johnson & Johnson Innovative Medicine (Janssen Pharmaceuticals website, October 2023)
- 21 Aug 2023 Phase-III clinical trials in Retinitis pigmentosa (In adults, In adolescents, In children, In the elderly) in Japan (Intraocular) (NCT05926583)
- 07 Jul 2023 Janssen Pharmaceutical plans a phase III trial for Retinitis Pigmentosa (In Children, In adolescents, In adults, In the elderly) in Japan (Intraocular, Injection) (NCT05926583)