Alexandria Forbes, PhD, President and CEO Professor Michel Michaelides, BSc, MB BS, MD(Res), FRCOphth, FACS Professor of Ophthalmology & Consultant Ophthalmic Surgeon at UCL Institute of Ophthalmology and Moorfields Eye Hospital. AAV-AIPL1 for the Treatment of Leber Congenital Amaurosis 4 (LCA4) Retinal Dystrophy AAV-AIPL1 for treatment of LCA4 genetic blindness 2 • MeiraGTx's rAAV8.hRKp.AIPL1 delivers the wild-type human AIPL1 gene under control of a photoreceptor- specific rhodopsin kinase promoter, hRK. ...

Goldman Sachs 45th Annual Global Healthcare Conference Optimized End-to-End in Genetic Medicine Future focus on in vivo delivery of biologic therapeutics for large indications and unmet needs Diverse Clinical Pipeline 3 late stage clinical programs pivotal/Phase 3 For prevalent non-inherited indications End-to-end GMP manufacturing Flexible and Scalable Next Generation Vector Optimization Potency, safety, dose, CofG Transformative RiboswitchTechnology In vivo delivery via oral small molecule • Retinitis Pig ...

An updated edition of the May 21, 2025, article.The promising field of genomics has undergone rapid evolution over the past decade. Genomics includes the study of a complete set of genes, their work process and way of interacting with each other and the environment. The primary focus is to evaluate all the genes of an organism rather than individual genes.This genetic information is being used to evaluate individual responses to drugs, leading to the development of targeted therapies. This, in turn, has pav ...

Multiple Poster Presentations Highlight the Depth and Novelty of MeiraGTx’s Technology Platforms for Gene and Cell TherapyLONDON 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 avai ...

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. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -34.21%. A quarter ago, it was expected that this company would post a loss of $0.48 per share when it actually produced a loss of $0.50, delivering a surprise of -4.17%.Over the last four quarters, the company has n ...

Table of Contents ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-38520 MeiraGTx Holdings plc UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) (Exact Name of Registrant as Specified in its Charter) Cayman Islands 98-1448305 ...

Exhibit 99.1 MeiraGTx Reports First Quarter 2025 Financial and Operational Results - 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 ...

- 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 gen ...

Dublin, April 29, 2025 (GLOBE NEWSWIRE) -- The "Cone Rod Dystrophy Market: Analysis By Type, By Treatment Type, By End User, By Region Size and Trends - Forecast up to 2030" has been added to ResearchAndMarkets.com's offering. Cone rod dystrophy represents a group of genetic eye disorders impacting the retina's cone and rod cells. The market for treating this condition is witnessing substantial growth, projected to rise from a value of US$131.29 million in 2024 to US$177.59 million by 2030. This growth is ...

I used to cover MeiraGTx (NASDAQ: MGTX ) a few years ago, but haven’t looked at it since 2022. MGTX develops genetic therapies for ocular diseases, Parkinson’s Disease and xerostomia. Lead asset is AAV-AQP1 targeting Xerostomia in a pivotal phase 2About the TPT ServiceThanks for reading. At the Total Pharma Tracker, we offer the following:-Our Android app and website features a set of tools for DIY investors, including a work-in-progress software where you can enter any ticker and get extensive curated rese ...