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Voretigene neparvovec - Spark Therapeutics

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Drug Profile

Voretigene neparvovec - Spark Therapeutics

Alternative Names: AAV2 hRPE65v2; AAV2-RPE65v2; Leber congenital amaurosis gene therapy; LTW 888; LUXTURNA; Luxturna; SPK-RPE65; voretigene neparvovec-rzyl

Latest Information Update: 19 Jun 2026

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At a glance

  • Originator The Childrens Hospital of Philadelphia
  • Developer Novartis; Spark Therapeutics
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    Yes - Leber congenital amaurosis; Retinitis pigmentosa
  • New Molecular Entity No

Highest Development Phases

  • Marketed Leber congenital amaurosis; Retinal dystrophies

Most Recent Events

  • 19 May 2026 Novartis Pharmaceuticals completes a phase III trial in Retinal dystrophies (In adults, In adolescents, In children, In the elderly) in Japan (Subretinal, Injection) (NCT04516369)
  • 30 Aug 2023 Launched for Retinal dystrophies (In adolescents, In children, In adults) in Japan (Subretinal, Injection)
  • 26 Jun 2023 Preregistration for Retinal dystrophies (In adolescents, In children, In adults) in Japan, prior to June 2023 (Subretinal, Injection)

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