MeiraGTx LLC

MeiraGTx presented four posters at ASGCT 2025, showcasing advancements in gene therapies for severe pediatric obesity, ALS, and frontotemporal dementia, highlighting their innovative approach to addressing significant unmet medical needs.

• MeiraGTx has secured a strategic collaboration with Hologen AI that includes a $200 million upfront payment and the formation of a joint venture with an additional $230 million in committed capital to advance AAV-GAD gene therapy for Parkinson's disease. • The partnership creates the first neuro-AI clinical drug development company, combining MeiraGTx's gene therapy expertise with Hologen's AI technology to transform CNS drug development for neurodegenerative and neuropsychiatric disorders. • Recent clinical data showed AAV-GAD therapy demonstrated significant benefits in Parkinson's disease patients, with meaningful improvements in motor function (UPDRS Part 3) and quality of life measures (PDQ-39).

Mass General Brigham's corneal stem cell therapy trial demonstrates positive outcomes across all enrolled patients, marking a significant advancement in regenerative eye treatment.

A groundbreaking gene therapy developed by Moorfields Eye Hospital and MeiraGTx has successfully restored vision in children born with LCA-AIPL1, a severe inherited eye disorder affecting 2-3 in 10 million newborns.

MeiraGTx's AAV2-hAQP1 gene therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA for grade 2/3 radiation-induced xerostomia (RIX).

AAV-GAD gene therapy demonstrated safety and improved motor scores by boosting GABA levels in the subthalamic nucleus in a Parkinson's disease trial.

MeiraGTx's AAV-GAD gene therapy met its primary endpoint, demonstrating safety and tolerability in a Phase I/II trial for Parkinson's disease.

• Pfizer has invested $49 million in Triana Biomedicines to discover novel molecular glue degraders for oncology, with Pfizer holding an exclusive option to license promising candidates. • Jazz Pharmaceuticals' Zepzelca, combined with Roche's Tecentriq, demonstrated improved overall survival and progression-free survival in small cell lung cancer patients in the IMforte Phase 3 study. • MeiraGTx's experimental gene therapy for Parkinson's disease met its primary endpoint in a small clinical trial, showing safety and tolerability, with encouraging signs of efficacy. • Akeso secured $250 million in private funding to advance global trials of its antibody drugs for cancer and autoimmune diseases, following positive data comparing its drug to Merck's Keytruda.