MeiraGTx Holdings plc

MeiraGTx Holdings plc is a clinical-stage biotechnology company developing gene therapies and cell therapies for serious genetic and neurodegenerative diseases. The company's pipeline centers on adeno-associated virus (AAV) based treatments targeting inherited retinal diseases, neurodegenerative conditions, and other genetic disorders. Key programs in clinical development include AAV-RPE65 for RPE65-associated retinal dystrophy, AAV-CNGB3 and AAV-CNGA3 for achromatopsia, and AAV-GAD for Parkinson's disease, all in Phase 2 trials. The company also has AAV-AQP1 in Phase 2 development for radiation-induced xerostomia and Phase 1/2 trials for Sjögren's syndrome. Additional preclinical programs address retinal dystrophy variants, Bardet-Biedl syndrome, Leber congenital amaurosis, and amyotrophic lateral sclerosis.

Beyond its gene therapy focus, MeiraGTx operates platform technologies including a riboswitch platform for metabolic peptide delivery and cell therapies targeting oncology, autoimmune disease, and chronic pain applications. The company maintains strategic collaborations with Johnson & Johnson Innovative Medicine for inherited retinal disease gene therapies and with Hologen Limited for CNS-directed genetic medicines development. Headquartered in New York, MeiraGTx operates with approximately 409 full-time employees and maintains a clinical-stage business model with no disclosed commercial revenue streams.