About MeiraGTx Holdings plc - Ordinary Shares (MGTX)
Meiragtx Holdings Plc is a biotechnology company focused on developing innovative genetic therapies to address significant unmet medical needs. The company leverages its advanced gene editing technology to create treatments for various rare and serious diseases, particularly those affecting the eye and central nervous system. By harnessing the power of gene therapy, Meiragtx aims to provide long-lasting solutions that target the underlying causes of genetic disorders, with a commitment to improving patient outcomes and quality of life. Through its research and clinical programs, the company is dedicated to pioneering advancements in the field of genetic medicine. Read More
- Announced strategic collaboration with Hologen AI, including a $200 million cash upfront payment to MeiraGTx and the formation of a joint venture, Hologen Neuro AI Ltd, with a further $230 million in capital committed to initially focus on expediting Phase 3 clinical development of AAV-GAD for Parkinson’s disease
- This RMAT designation is based on data from 3 clinical studies demonstrating the potential benefit of AAV-GAD as a one-time treatment for Parkinson’s disease
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- Today announced strategic collaboration with Hologen AI, including a $200 million upfront payment to MeiraGTx and the formation of a joint venture, Hologen Neuro AI Ltd, with a further $230 million in capital committed to initially focus on expediting Phase 3 clinical development of AAV-GAD for Parkinson’s disease
LONDON and NEW YORK, Feb. 21, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced the publication of results from the first-in-human interventional study to treat children with AIPL1-associated severe retinal dystrophy. The data were published in The Lancet in a paper titled, "Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study". This paper presents data from the first 4 children treated unilaterally with rAAV8.hRKp.AIPL1. A further 7 children with LCA4 were treated bilaterally with rAAV8.hRKp.AIPL1 demonstrating similarly remarkable effects on vision, with all 11 of the 11 children treated who were blind at birth now having visual acuity in the treated eyes.
MeiraGTx has recently received Rare Pediatric Disease Designation (RPDD) for four inherited retinal diseases (IRDs) reflecting the transformative therapeutic potential of the Company’s proprietary technology platforms
- RMAT designation recognizes the preliminary clinical evidence of the potential benefit of AAV2-hAQP1 as a one-time treatment for this debilitating condition
LONDON and NEW YORK, Nov. 13, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced financial and operational results for the third quarter ended September 30, 2024, and provided a corporate update. The Company also announced that following meetings with the UK Medicines and Healthcare products Regulatory Agency (MHRA), the Company intends to submit a Marketing Authorization Application under exceptional circumstances for AAV-AIPL1 in the United Kingdom without the need for further clinical studies. The Company is also initiating discussions with the FDA around the potential for a similar pathway to approval in the U.S. In addition, MeiraGTx announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the U.S. Food and Drug Administration (FDA) have granted the Company three Rare Pediatric Disease Designations to its AAV8-RK-AIPL1 program, AAV8-RK-BBS10 program, and AAV5-RDH12 program, each for the treatment of inherited retinal diseases.
MeiraGTx's clinical study of AAV-GAD for Parkinson's disease showed significant improvements in motor function and quality of life in high- and low-dose groups, with no serious adverse events. Participants will be followed for five years in a long-term study.
