MeiraGTx Holdings plc is a clinical stage genetics medicines company focusing on developing treatments for patients with serious diseases. The company develops various therapies for ocular diseases, including inherited retinal diseases and large degenerative ocular diseases; neurodegenerative diseases; and xerostomia.
It is also developing AAV-AQP1 in a Phase 2 trial for radiation-induced grade 2/3 xerostomia, as well as in a Phase 1/2 trial for Sjogren's syndrome. Additionally, AAV-GAD is in a Phase 2 trial for Parkinson's disease, while AAV-RPE65 is in a Phase 2 trial for RPE65-associated retinal dystrophy. AAV-CNGB3 and AAV-CNGA3 are both in Phase 2 trials for achromatopsia, and the company offers AAV-AIPL1 for Leber congenital amaurosis (LCA) 4.
In addition, the company's products under preclinical development include AAV-RDH12 for retinol dehydrogenase 12 mutation-associated retinal dystrophy; AAV-BBS10 for Bardet-Biedl syndrome (BBS) due to BBS10 mutations; AAV-RetGC for LCA type 1; and AAV-UPF1 for amyotrophic lateral sclerosis. The company also has programs for Alzheimer's disease and wet and dry neovascular age-related macular degeneration.
Furthermore, it provides a riboswitch platform for the delivery of metabolic peptides and cell therapies for oncology, autoimmune disease, and long-term intractable pain.
MeiraGTx has collaborations with Johnson & Johnson Innovative Medicine for the research, development, and commercialization of gene therapies for the treatment of inherited retinal disease. It also collaborates with Hologen Limited for the research, development, manufacture, and commercialization of AAV-GAD investigational gene therapy for the treatment of Parkinson's disease, AAV-BDNF investigational gene therapy for genetic obesity disorders, and other genetic medicines to the central nervous system.
MeiraGTx Holdings plc was founded in 2015 and is based in New York, New York.
