Laruparetigene zosaparvovec - Beacon Therapeutics
Alternative Names: AGTC-501; BIIB-088; rAAV2tYF-GRK1-RPGRLatest Information Update: 18 Jun 2024
At a glance
- Originator Applied Genetic Technologies Corporation
- Developer Beacon Therapeutics
- Class Eye disorder therapies; Gene therapies
- Mechanism of Action Gene transference; RPGR protein modulators
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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 II/III Retinitis pigmentosa
Most Recent Events
- 12 Jun 2024 AGTC 501 (laruparetigene zovaparvovec) receives Innovative Medicine Designation (ILAP) status in the UK for X-linked retinitis pigmentosa
- 12 Jun 2024 Laruparetigene zosaparvovec - Beacon Therapeutics receives Fast Track designation for Retinitis pigmentosa [Intraocular,Injection] (In adolescents, In children, In adults) in USA
- 12 Jun 2024 Laruparetigene zosaparvovec - Beacon Therapeutics receives Priority Medicine (PRIME) status for Retinitis pigmentosa in European Union