Inherited Retinal Disease

Gene therapy appears safe, effective in X-linked retinitis pigmentosa

Primary results from the Phase 1/2 study evaluating the investigational gene therapy botaretigene sparoparvovec in patients with the inherited retinal disease X-linked retinitis pigmentosa (XLRP) associated with the retinitis pigmentosa GTPase regulator (RPGR) gene were presented at the AAO 2022 Annual meeting. Treatment with the investigational gene therapy was found...