Press release
MeiraGTx Presents Clinical Data on Botaretigene Sparoparvovec for the Treatment of X-Linked Retinitis Pigmentosa at the Association for Research in Vision and Ophthalmology 2022 Annual Meeting
LONDON and NEW YORK, May 04, 2022 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical-stage gene therapy company, today
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[{"type":"text","content":"LONDON and NEW YORK, May 04, 2022 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical-stage gene therapy company, today announced that additional clinical data from the Phase 1/2 trial of botaretigene sparoparvovec for the treatment of X-linked Retinitis Pigmentosa (XLRP) were presented at the Association for Research in Vision and Ophthalmology 2022 Annual Meeting (ARVO), in Denver, Colorado by Professor Michel Michaelides. A poster presentation of human retinal organoid vector efficacy data was also presented at the conference. Oral Presentation Details: Abstract Title: AAV5-RPGR (botaretigene sparoparvovec) Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) Demonstrates Localized Improvements in Static PerimetryPresenter: Dr. Michel Michaelides, Professor of Ophthalmology, UCL Institute of Ophthalmology in Dept. of GeneticsDate and Time: Wednesday, May 4, 2022, at 1:38 pm MDT (3:38 pm EDT) Patients in the multicenter, open-label Phase 1/2 trial were given botaretigene sparoparvovec subretinally at 1 of 3 doses to the worse-seeing eye. Changes in retinal sensitivity and the volumetric analysis of the central 30 degrees of the retinal field were examined and compared to the untreated surrounding area as well as the retina of the untreated fellow eye. Exploration of the association between the location of botaretigene sparoparvovec delivery and changes in retinal sensitivity was undertaken by overlaying bleb topography onto sensitivity heat maps. Data from the study indicates improvements in photoreceptor function assessed through 12 months post-treatment suggesting local efficacy of botaretigene sparoparvovec gene therapy in XLRP patients. The treatment effect is observed within the treated bleb and may also extend beyond the margins of the bleb following surgery owing to subretinal extension before retinal reattachment. Poster Presentation Details: Abstract Title: AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa (XLRP): Human retinal organoid vector efficacy dataPresenting Author: Paul E. Sladen Mutations within the retinitis pigmentosa GTPase regulator (RPGR) are the most frequent cause of (XLRP). This study investigated the efficacy of RPGRORF15 gene supplementation in human RPGR-deficient retinal organoids (ROs). Successful differentiation of RPGR-KO iPSCs was...