cevaretigene ritoparvovec

Cevaretigene ritoparvovec is an investigational gene therapy designed to treat inherited retinal diseases caused by biallelic mutations in the RPE65 gene, such as Leber congenital amaurosis. It uses an adeno-associated virus (AAV) vector—specifically, a hybrid serotype 2/5 (AAV2/5)—to deliver a functional copy of the human RPE65 gene to retinal cells. The therapy aims to restore visual function by enabling production of the missing or defective enzyme in patients with these genetic mutations. Cevaretigene ritoparvovec is being developed primarily by MeiraGTx, with contributions from University College London and the Medical Research Council[2][3][4]. As of early 2025, it has reached Phase I/II clinical trials for Leber congenital amaurosis and holds orphan drug status for this indication[2].

Other names

RPE65-gene-therapy-MeiraGTxRPE-65-gene-therapy-MeiraGTxRPE 65-gene-therapy-MeiraGTx