MeiraGTx Holdings plc is a clinical-stage genetic medicines company focused on developing treatments for patients with serious diseases. Its pipeline includes therapies for ocular diseases such as inherited retinal diseases and large degenerative ocular diseases, neurodegenerative diseases, and xerostomia. The company is developing AAV-AQP1 for radiation-induced grade 2/3 xerostomia and Sjögren's syndrome; AAV-GAD in Phase 2 for Parkinson's disease; AAV-RPE65 in Phase 2 for RPE65-associated retinal dystrophy; and AAV-CNGB3 and AAV-CNGA3 in Phase 1/2 for achromatopsia, as well as AAV-AIPL1 for Leber congenital amaurosis (LCA) 4. Preclinical programs include AAV-RDH12 for retinol dehydrogenase 12 mutation-associated retinal dystrophy; AAV-BBS10 for Bardet-Biedl syndrome due to BBS10 mutations; AAV-RetGC for LCA type 1; and AAV-UPF1 for amyotrophic lateral sclerosis, along with programs for Alzheimer's disease and wet and dry neovascular age-related macular degeneration. It also provides a riboswitch platform for the delivery of metabolic peptides and cell therapies for oncology, autoimmune disease, and long-term intractable pain. The company has collaborations with Johnson & Johnson Innovative Medicine for gene therapies for inherited retinal disease, and with Hologen Limited for AAV-GAD for Parkinson's disease, AAV-BDNF for genetic obesity disorders, and other genetic medicines for the central nervous system. MeiraGTx Holdings plc was founded in 2015 and is based in New York, New York.