MeiraGTx Holdings

MGTX

Market cap $623M

LONDON and NEW YORK, Oct. 07, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the Company will exhibit two posters at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress, which is being held from October 7-10, 2025, in Seville, Spain. The posters are available on the Posters and Publications page of the Company's website.

MeiraGTx is a clinical-stage gene therapy biotech with end-to-end GMP. Its late-stage assets include AAV-AIPL1, AAV-AQP1, and Bota-Vec. AAV-AQP1 is in pivotal Phase 2 trials under the FDA's RMAT designation for radiation-induced xerostomia. MGTX anticipates its BLA by 2026. I believe their Hologen partnership for PD is MGTX's main value driver. This should fund AAV-GAD through Phase 3 and commercialization if successful.

MeiraGTx Holdings PLC (MGTX) came out with a quarterly loss of $0.48 per share versus the Zacks Consensus Estimate of a loss of $0.52. This compares to a loss of $0.76 per share a year ago.

-  Gained alignment with U.S. Food and Drug Administration (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 Biologics License Application (BLA) filing; on track for potential data readout late 2026 -  FDA Granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD for the treatment of Parkinson's disease -  In collaboration with Hologen, FDG-PET data from positive Phase 2 double-blind, sham-surgery controlled clinical trials of AAV-GAD shows significant disease-modifying effects in pathological basal ganglia circuitry, including the substantia nigra – the site of dopamine-producing neurons affected in Parkinson's disease -  On track to file for Marketing Authorization Approval (MAA) under exceptional circumstances with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of LCA4, and BLA in the US with the FDA via a similar pathway to approval in the fourth quarter of 2025 LONDON and NEW YORK, Aug. 14, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced financial and operational results for the second quarter ended June 30, 2025, and provided a corporate update. “During the second quarter, we continued to have very productive regulatory interactions with the MHRA and FDA around multiple later stage clinical programs as well as manufacturing,” said Alexandria Forbes, Ph.D.

Investors interested in the Genomics and Synthetic Biology theme should keep an eye on MGTX, BEAM and KRYS.

Multiple Poster Presentations Highlight the Depth and Novelty of MeiraGTx's Technology Platforms for Gene and Cell Therapy Multiple Poster Presentations Highlight the Depth and Novelty of MeiraGTx's Technology Platforms for Gene and Cell Therapy

MeiraGTx Holdings PLC (MGTX) came out with a quarterly loss of $0.51 per share versus the Zacks Consensus Estimate of a loss of $0.38. This compares to loss of $0.77 per share a year ago.

-  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 BLA filing   -  Efficacy data of rAAV8.hRKp.AIPL1 for the treatment of AIPL1-related retinal dystrophy, or LCA4, published in The Lancet, demonstrating meaningful responses in 11 out of 11 LCA4 children treated. Filing for Marketing Authorization Approval (MAA) under exceptional circumstances with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and in discussions with the FDA around a potentially similar pathway to approval in the US LONDON and NEW YORK, May 13, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced financial and operational results for the first quarter ended March 31, 2025, and provided a corporate update.

-  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 treatment of Parkinson's disease not adequately controlled with anti-Parkinsonian medications. This RMAT was awarded following the presentation to the FDA of positive data from 3 clinical studies demonstrating the benefit of AAV-GAD when administered in a one-time stereotactic infusion to the subthalamic nucleus in the brain.

The global cone rod dystrophy market, valued at US$131.29 million in 2024, is projected to reach US$177.59 million by 2030, growing at a CAGR of 5.33%. Key drivers include advancements in gene therapies, personalized medicine, and government programs. North America remains the largest market, while Asia Pacific leads growth. Major players like SparingVision and Beacon Therapeutics propel innovation through ongoing clinical trials. The global cone rod dystrophy market, valued at US$131.29 million in 2024, is projected to reach US$177.59 million by 2030, growing at a CAGR of 5.33%. Key drivers include advancements in gene therapies, personalized medicine, and government programs. North America remains the largest market, while Asia Pacific leads growth. Major players like SparingVision and Beacon Therapeutics propel innovation through ongoing clinical trials.