MeiraGTx is a clinical-stage gene therapy company developing treatments for diseases affecting the eye, salivary gland, and brain. The company uses adeno-associated virus (AAV) vectors to deliver genetic material directly to target tissues, enabling low doses and avoiding the systemic immune responses that can complicate gene therapy. MeiraGTx has no approved products. Its most advanced programs are AAV-hAQP1 for radiation-induced dry mouth (Breakthrough Therapy Designation; Phase 2 pivotal-track trial underway) and AAV-GAD for Parkinson's disease (FDA RMAT designation), which delivers a gene to the brain via a one-time infusion to restore normal motor circuitry. MeiraGTx also has an ophthalmology pipeline targeting inherited retinal dystrophies, including AAV-AIPL1 for LCA4, which it licensed to Eli Lilly in late 2025 for up to $400M+ in milestones plus royalties. The company funds operations through licensing deals with Johnson & Johnson, Lilly, and Hologen, as well as contract manufacturing from its GMP facilities in the UK and Ireland. MeiraGTx also owns a riboswitch platform that uses an oral small molecule to switch gene expression on or off, enabling dose-responsive control of a delivered transgene. Applications include metabolic diseases (GLP-1, leptin), CAR-T cell therapy, and gene editing. Sanofi and Lilly hold rights of first negotiation to use the riboswitch in select therapeutic areas.