Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa.
Michel MichaelidesCagri G BesirliYesa YangThales A C DE GuimaraesSui Chien WongRachel M HuckfeldtJason I ComanderJosé-Alain SahelSyed Mahmood ShahJames J L TeeNeruban KumaranAnastasios GeorgiadisPansy MinnickRobert ZeldinStuart NaylorJialin XuMichael ClarkEddy AngladePeggy WongPenny R FleckAlbert FungColleen PelusoAngelos KalitzeosMichalis GeorgiouCaterina RipamontiAlexander J SmithRobin R AliAlexandria ForbesJames BainbridgePublished in: American journal of ophthalmology (2024)
AAV5-hRKp.RPGR demonstrated an anticipated and manageable AE profile through 52 weeks. Safety and efficacy findings support investigation in a phase 3 trial.
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