Press release
MeiraGTx Reports Third Quarter 2022 Financial and Operational Results and Receives $25 Million Investment from Johnson & Johnson Innovation - JJDC, Inc.
-- Updated Positive Topline Data from the Phase 1/2 Trial of Botaretigene Sparoparvovec (AAV-RPGR) for the Treatment of X-linked Retinitis Pigmentosa (XLRP)

About this update from Meiragtx Holdings Plc
[{"type":"text","content":"-- Updated Positive Topline Data from the Phase 1/2 Trial of Botaretigene Sparoparvovec (AAV-RPGR) for the Treatment of X-linked Retinitis Pigmentosa (XLRP) -- Now Dosing Patients in Phase 1 Trial Evaluating AAV-GAD Gene Therapy for Parkinson’s Disease -- Received $25.0 Million Investment from Johnson & Johnson Innovation – JJDC, Inc. (“JJDC”), the Investment Arm of Johnson and Johnson -- Company to Provide Clinical Program Update for the Phase 1 AQUAx Trial of AAV-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia in December 2022 LONDON and NEW YORK, Nov. 10, 2022 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage gene therapy company, today announced financial results for the third quarter ended September 30, 2022, and provided an update on recent progress. “As we head into year-end, we continue to make progress in advancing our clinical and preclinical pipeline,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “This includes presenting data from our Phase 1/2 clinical study in XLRP at AAO, further demonstrating botaretigene sparoparvovec’s potential to improve vision in patients with retinitis pigmentosa. We are currently dosing patients in the Phase 3 LUMEOS trial and remain on track for a BLA in 2024. We are also pleased with the $25 million investment from JJDC which will help us to further our programs and continue development of our internal capabilities.” “In addition, we are now dosing patients with adeno-associated virus (AAV) encoding glutamic acid decarboxylase (AAV-GAD), an investigational gene therapy product candidate for Parkinson’s disease,” Dr. Forbes continued. “And in our xerostomia program, we look forward to presenting updated data from our Phase 1 AQUAx trial later this year.” “We are also very excited by the progress of our technology and manufacturing platforms,” stated Dr. Forbes. “We published 15 abstracts at ESGCT in October this year, highlighting the robustness of our riboswitch gene regulation technology for gene and cell therapies, as well as the depth of our proprietary promoter and manufacturing and process development platforms.” Recent Highlights and Anticipated Milestones Botaretigene Sparoparvovec for the Treatment of XLRP: In October 2022, MeiraGTx presented positive data from the MGT009 Phase ...