botaretigene sparoparvovec

Botaretigene sparoparvovec is an investigational gene therapy designed to treat X-linked retinitis pigmentosa (XLRP) caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. It uses an adeno-associated virus vector type 5 (AAV5) to deliver a functional copy of the RPGR gene directly into retinal cells via subretinal injection. The goal is to enable affected retinal cells to produce healthy RPGR protein, thereby improving or stabilizing visual function and slowing disease progression. Botaretigene sparoparvovec has received Orphan Drug and Fast Track designations from the FDA, as well as PRIME and ATMP status from the EMA. It is being developed through a collaboration between Johnson & Johnson Innovative Medicine and MeiraGTx, with University College London as the originator[1][3][4][6].

Other names

RPGR AAV gene therapy