MeiraGTx Holdings plc
NASDAQ Global SelectCapital Structure
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in mil. unless spec.Working Capital
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in mil. unless spec.Quarterly Earnings Per Share
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in mil. unless spec.Company Description
- CEO
- Alexandria Forbes
- Full Time Employees
- 393
- Sector
- Healthcare
- Industry
- Biotechnology
- Address
- 655 Third Avenue New York City NY United States of America 10017
- IPO Date
- Jun 8, 2018
- CIK
- 0001735438
- Website
- meiragtx.com
- Similar Companies
- Business
- MeiraGTx Holdings plc (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicines company focused on developing novel gene therapies for rare and prevalent diseases in ophthalmology, salivary gland disorders, neurodegenerative conditions, and metabolic diseases. Founded in 2015 and headquartered in New York, New York, with additional manufacturing facilities in the United Kingdom and Ireland, the company advances a broad pipeline supported by proprietary viral vector optimization, end-to-end GMP manufacturing capabilities, and riboswitch gene regulation technology for precise, dose-responsive control of gene expression via oral small molecules. Core programs include AAV-AQP1 for radiation-induced xerostomia (Phase 3-ready) and Sjogren’s disease (preclinical); AAV-GAD for Parkinson’s disease (Phase 3-ready, with FDA RMAT designation); botaretigene sparoparvovec (AAV-RPGR) for X-linked retinitis pigmentosa (Phase 3 completed, subject to asset purchase agreement with Janssen); AAV-AIPL1 for AIPL1-associated Leber congenital amaurosis 4 (specials license in the UK, pursuing expedited approvals); preclinical ophthalmology candidates such as AAV-RDH12 for RDH12-associated retinal dystrophy, AAV-ABCA4 for Stargardt’s disease, AAV-VEGFR2 for wet AMD, and undisclosed programs for dry AMD/GA and Bardet-Biedl syndrome; riboswitch-regulated therapies including RiboLeptin for lipodystrophies, undisclosed candidates for intractable neuropathic pain and GLP-1/GIP combinations for obesity/MASH/metabolic disease, and Ribo-CAR-T for oncology and autoimmune diseases; as well as AAV-BDNF for MC4R/BDNF genetic obesity and preclinical neurodegenerative programs like AAV-UPF1/AAV-CNTFR for ALS. Geographically, MeiraGTx operates primarily in the United States, United Kingdom, and European Union, targeting patients with serious unmet needs in these therapeutic areas. Recent developments include a March 2025 strategic collaboration with Hologen AI featuring a $200 million upfront payment and formation of Hologen Neuro AI Ltd joint venture with up to $230 million committed capital to accelerate Phase 3 development of AAV-GAD for Parkinson’s disease, industrialize manufacturing, and advance CNS programs; FDA RMAT designations for AAV-GAD (May 2025) and AAV2-hAQP1 for xerostomia (January 2025); publication of landmark efficacy data in The Lancet for AAV-AIPL1 restoring vision in 11 children blind from birth (February 2025), prompting MAA filing in the UK and U.S. discussions; alignment with FDA on the pivotal Phase 2 AQUAx2 study for xerostomia (2025); an asset purchase agreement with Janssen for up to $415 million covering botaretigene sparoparvovec (announced earlier); and multiple Rare Pediatric Disease Designations for inherited retinal programs.
Company News
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