MeiraGTx Holdings plc, a clinical stage gene therapy company, focusing on developing treatments for patients living with serious diseases. The company develops various therapies for ocular diseases, including inherited blindness, as well as Xerostomia following radiation treatment for head and neck cancers; degenerative diseases; neurodegenerative diseases, such as amyotrophic lateral sclerosis; and Parkinson's diseases. Its programs in clinical development include Phase 1/2 clinical stage programs in Achromatopsia, X-Linked Retinitis Pigmentosa, RPE65-deficiency, and radiation-induced Xerostomia, as well as Parkinson's program. The company also focuses on initiating a clinical program in xerostomia related to Sjogren's syndrome and have preclinical programs in neurodegenerative diseases. It has a research collaboration agreement with Janssen Pharmaceuticals, Inc. to develop regulatable gene therapy treatment using the company's proprietary riboswitch technology. The company was incorporated in 2015 and is based in New York, New York.
IPO Year: 2018
Exchange: NASDAQ
Website: meiragtx.com
| Date | Price Target | Rating | Analyst |
|---|---|---|---|
| 7/31/2024 | $36.00 | Buy | Chardan Capital Markets |
LONDON and NEW YORK, May 13, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical stage genetic medicines company, today announced the Company will exhibit four poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting, which is being held from May 13-17, 2025, in New Orleans, LA. The posters will be available on the Posters and Publications page of the Company's website. The details of the poster presentations are below: Poster 507: An Ultra-Low Dose of a Localized CNS Gene Therapy for Severe Pediatric ObesityPoster Session: Tuesday, May 13, 6:00-7:30 pm CDTAbstract:Brain-derived neurotrophic factor (BDN
- 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 - U.S. Food and Drug Administration (FDA) Granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD for the treatment of Parkinson's disease - Gained alignment with FDA on the ongoing Phase 2 AQUAx2 randomized double-blind, placebo-controlled pivotal study in Grade 2/3 radiation-induced xerostomia (RIX) to support a potential BL
- 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 - RMAT designation includes the benefits of the Fast Track and Breakthrough Therapy designations, allows frequent regulatory interactions with the FDA, and potential routes to accelerated approval and Priority Review LONDON and NEW YORK, May 09, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV-GAD for the t
- 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 - MeiraGTx and Hologen have created the first neuro-AI clinical drug development company to transform the discovery and development of therapies targeting CNS circuitry in neurodegenerative and neuropsychiatric disorders - Announced positive data from randomized, double blind, sham-controlled clinical bridging study of AAV-GAD for the treatment of Parkinson's disease demonst
- MeiraGTx to receive $200 million in upfront cash consideration - MeiraGTx and Hologen will form a joint venture, Hologen Neuro AI Ltd, with an additional $230 million committed capital from Hologen to fund 100% of the development of AAV-GAD for Parkinson's disease through to commercialization, as well as other potential pipeline products - MeiraGTx will enter into clinical and commercial supply agreements with Hologen Neuro AI Ltd to manufacture AAV-GAD and other locally delivered CNS genetic medicines - Hologen will also fund a portion of MeiraGTx's manufacturing operations and will own a minority stake in MeiraGTx's manufacturing subsidiary LONDON and NEW YORK, March 13,
As disclosed in the paper, 4 out of 4 young children with the AIPL1-related retinal dystrophy, LCA4, benefited substantially from unilateral subretinal administration of rAAV8.hRKp.AIPL1 with improved visual acuity, functional vision, and protection against progressive retinal degenerationFollowing the strong safety and substantial efficacy demonstrated in this first cohort of 4 children treated unilaterally, a further 7 children have now been treated bilaterally and all showed substantial benefit from treatment with rAAV8.hRKp.AIPL1Meaningful responses have been observed in 11 out of 11 LCA4 children treated to date with rAAV8.hRKp.AIPL1All 11 children treated with rAAV8.hRKp.AIPL1 between
LONDON and NEW YORK, Jan. 22, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings Plc (NASDAQ:MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the U.S. Food and Drug Administration (FDA) has granted the Company Rare Pediatric Disease Designation to its AAV8-RK-RetGC program for the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1). This is the fourth Rare Pediatric Disease Designation the Company has received in the last three months, including AAV8-RK-AIPL1 for the treatment of LCA4 retinal dystrophy, AAV8-RK-BBS10 for the treatment of Bardet-Biedl syndrome (BBS) due to BBS10 mutations and AAV5-RDH12 for the treatment of R
- RMAT designation recognizes the preliminary clinical evidence of the potential benefit of AAV2-hAQP1 as a one-time treatment for this debilitating condition - RMAT designation includes the benefits of the Fast Track and Breakthrough Therapy designations, allows frequent regulatory interactions with the FDA, and potential routes to accelerated approval and Priority Review LONDON and NEW YORK, Dec. 09, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical stage genetic medicines company, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV2-hAQP1 for the tr
ROCKVILLE, Md. and SUZHOU, China, Nov. 24, 2024 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in discovering, developing and commercializing therapies to address global unmet medical needs primarily for malignancies, today announced that it has appointed Ms. Marina S. Bozilenko and Dr. Debra Yu as additional independent non-executive directors of the company with effect from November 25, 2024. "I would like to extend a warm welcome to Ms. Marina S. Bozilenko and Dr. Debra Yu, who are joining Ascentage Pharma as independent non-executive
Received 3 Rare Pediatric Disease Designations (RPDD) from FDA for each of 3 potential therapies for 3 different rare inherited retinopathies including AAV-AIPL1 Agreed on pathway with MHRA for Marketing Authorization Application (MAA) under exceptional circumstances for AAV-AIPL1 for the treatment of Leber congenital amaurosis (LCA4) retinal dystrophy without further clinical studies Announced positive data from randomized, sham-controlled clinical bridging study of AAV-GAD for the treatment of Parkinson's disease LONDON and NEW YORK, Nov. 13, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical stage genetic medicines company, today announced
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