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Botaretigene sparoparvovec - Johnson & Johnson Innovative Medicine/MeiraGTx

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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; bota-vec; JNJ-74765340; RPGR AAV gene therapy

Latest Information Update: 23 Oct 2024

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At a glance

  • Originator University College London
  • Developer Johnson & Johnson Innovative Medicine; MeiraGTx
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Gene transference; RPGR protein replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Retinitis pigmentosa
  • New Molecular Entity No

Highest Development Phases

  • Phase III Retinitis pigmentosa

Most Recent Events

  • 11 Oct 2024 Janssen Research & Development plans a phase II trial for Retinitis Pigmentosa (In children, In adolescents, In adults, In the elderly) (Intraocular) in October 2024 (NCT06646289 )
  • 10 Oct 2024 Janssen Research & Development initiates a phase II trial for Retinitis Pigmentosa (In children, In adolescents, In adults, In the elderly) in USA (Intraocular) (NCT06646289 )
  • 30 Sep 2024 Janssen Research & Development completes a phase III trial in Retinitis pigmentosa (In children, In adolescents, In the elderly, In adults) in USA, United Kingdom, Canada, Denmark, Switzerland, Israel, the Netherlands, France, Spain, Ireland, Belgium, Germany and Italy (Intraocular) (NCT04671433)

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