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larupretigene zovaparvovec
- Development stage
- Unknown
- Lead developer
- Beacon Therapeutics
- Modality
- Gene Therapies
- Administration
- Ophthalmic
01
Overview
Larupretigene zovaparvovec (AGTC-501) is an investigational gene therapy developed for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the *RPGR* gene. The therapy utilizes a recombinant adeno-associated virus (rAAV) vector with a modified AAV2 capsid (AAV2tYF), which contains three surface tyrosine-to-phenylalanine mutations to enhance transduction efficiency. It delivers a codon-optimized human *RPGR* cDNA (hRPGRco) under the control of a photoreceptor-specific human G protein-coupled receptor kinase 1 (GRK1) promoter. Administered via subretinal injection, the product aims to restore functional RPGR protein expression in rod and cone photoreceptors, thereby slowing or preventing the progressive vision loss characteristic of XLRP. Originally developed by Applied Genetic Technologies Corporation (AGTC), the program is currently being advanced by Beacon Therapeutics.
- Other names
- larupretigene zovaparvovec
02
Targets
RPGR (Retinitis pigmentosa GTPase regulator)
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