botaretigene sparoparvovec (Janssen Pharmaceutical)

Botaretigene sparoparvovec is an investigational adeno-associated virus serotype 5 (AAV5) vector-based gene therapy designed for the treatment of X-linked retinitis pigmentosa (XLRP) associated with mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Developed through a collaboration between MeiraGTx and Janssen (Johnson & Johnson), the therapy delivers a functional copy of the human RPGR gene under the control of a human rhodopsin kinase (hRKp) promoter. It is administered via a one-time subretinal injection with the goal of preserving or potentially restoring vision by counteracting the progressive loss of retinal cells. While the therapy has received Fast Track and Orphan Drug designations from the FDA and PRIME designation from the EMA, it recently failed to meet the primary endpoint in the Phase 3 LUMEOS clinical trial.

Other names

botaretigene sparoparvovec-vzpz