MeiraGTx Holdings plc Aktie News

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 comme...

-  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-GA...

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

-  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 Advanc...

-  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 YOR...

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 innova...