MeiraGTx is a clinical-stage biotechnology company specializing in the development and commercialization of adeno-associated virus (AAV) gene therapies for rare diseases with high unmet medical need. Since its founding in 2014 as a spin-out from GlaxoSmithKline, the company has built a proprietary AAV platform designed to deliver therapeutic genes to target tissues with precision and durability. Its research and development efforts are focused on ophthalmology, central nervous system disorders, lysosomal storage diseases and oncology indications.
The company’s lead program, LUMEVOQ (AAV2-REP1), targets choroideremia, a rare inherited retinal degeneration, and has advanced through Phase II/III clinical studies. Additional programs leverage varying AAV serotypes and promoters to optimize tissue tropism, including candidates for X-linked retinitis pigmentosa, spinal muscular atrophy and hemophilia A. MeiraGTx also collaborates with academic institutions and biotech partners to expand its pipeline into new therapeutic areas and enhance manufacturing capabilities for clinical and commercial-scale AAV production.
Headquartered in London with a corporate presence in New York, MeiraGTx operates a fully integrated development and manufacturing infrastructure that spans discovery, preclinical research and regulatory engagement. The company has established GMP-compliant manufacturing suites in the United Kingdom and the United States to support its late-stage programs and prospective commercial launches. This integrated model aims to streamline process development and ensure timely supply of clinical material.
MeiraGTx is led by a management team and board of directors with extensive experience in gene therapy, regulatory affairs and biopharmaceutical commercialization. The leadership group combines expertise drawn from major pharmaceutical companies, academic research centers and specialized gene therapy organizations, positioning MeiraGTx to navigate the regulatory landscape and advance its pipeline toward potential regulatory approvals.
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