MeiraGTx: Phase 1/2 Data Show Safety, Vision Improvement in Retinitis Pigmentosa Gene Therapy

 MeiraGTx Holdings plc (Nasdaq:MGTX), a vertically integrated, clinical-stage gene therapy company, announced today the primary results from the Phase 1/2 study evaluating the investigational gene therapy botaretigene sparoparvovec (formerly AAV-RPGR) in patients with the inherited retinal disease X-linked retinitis pigmentosa (XLRP) associated with the retinitis pigmentosa GTPase regulator (RPGR) gene.

Treatment with botaretigene sparoparvovec was found to have an acceptable safety profile and efficacy assessments in this proof-of-concept study demonstrated improvements in retinal sensitivity, visual function and functional vision.¹ These findings were presented in a late-breaking oral presentation today at the Retina Subspecialty Day program of the American Academy of Ophthalmology (AAO) 2022 Annual Meeting (Abstract #30071754) by Professor Michel Michaelides.

https://www.marketscreener.com/quote/stock/MEIRAGTX-HOLDINGS-PLC-44154954/news/Late-Breaking-Phase-1-2-Data-Demonstrates-Safety-Profile-of-Investigational-Gene-Therapy-Botaretigen-41905060/