MeiraGTx Holdings plc

We are a vertically integrated, clinical-stage genetic medicines company. We have a broad pipeline of late-stage clinical programs, including treatments for radiation-induced xerostomia, Parkinson's disease, and AIPL1-associated retinal dystrophy. Our clinical programs use targeted local delivery of small doses of genetic medicines to treat both inherited and more common conditions with severe unmet need. Our internal end-to-end manufacturing capabilities support the development of our clinical pipeline. We operate two viral vector production facilities for good manufacturing practices (GMP), internal plasmid production for GMP, and an in-house Quality Control hub for stability and release. These facilities are suitable for Investigational New Drug application (IND) through commercial supply. We have also developed a proprietary manufacturing platform with industry-leading yield and quality aspects and commercial readiness. Our core capabilities in viral vector and capsid optimization enable increased potency, decreased dose, and significantly reduced cost of goods for our genetic medicines. We have developed a transformative gene regulation platform using bespoke synthetic riboswitch technology invented in-house. This technology allows for the precise, dose-responsive expression of any transgene under the control of oral small molecules. We are focusing the riboswitch platform on the in vivo delivery of biologic therapeutics such as the metabolic peptides glucagon-like peptide-1 (GLP-1), glucose-dependent insulinotropic polypeptide (GIP), glucagon, amylin, peptide YY (PYY), and leptin via oral small molecules. We are also applying it to cell therapy for oncology and autoimmune diseases, and long term intractable pain. We have developed unique comprehensive technology capabilities to apply genetic medicine to more prevalent diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high. We own and operate manufacturing facilities in London, United Kingdom and Shannon, Ireland.