laruparetigene zovaparvovec

Laruparetigene zovaparvovec is an investigational gene therapy developed for the treatment of X-linked retinitis pigmentosa (XLRP), a rare inherited retinal disorder that leads to progressive vision loss. The therapy uses an adeno-associated virus (AAV) vector to deliver a functional copy of the RPGR gene directly into retinal cells via subretinal injection. By expressing the full-length RPGR protein, it aims to address both rod and cone photoreceptor degeneration caused by mutations in the RPGR gene. Laruparetigene zovaparvovec is currently in Phase 3 clinical development and has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA as well as Fast Track status in the US and similar designations in Europe and the UK[1][3][5][6][7].

Brand names

laru-zova

Other names

laruparetigene zovaparvovecAGTC-501AGTC501AGTC 501laru-zova