LONDON and NEW YORK, Nov. 10, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced a broad strategic collaboration in the area of ophthalmology with Eli Lilly and Company (“Lilly”).
MeiraGTx will grant Lilly worldwide exclusive rights to its AAV-AIPL1 program for treatment of one of the most severe inherited retinopathies, Leber congenital amaurosis 4 (LCA4) owing to genetic deficiency of Aryl-hydrocarbon-interacting protein-like 1 (AIPL1). Clinical data from 11 children under the age of 4 who were born legally blind as a result of mutations in the AIPL1 gene were unprecedented, with all 11 blind children gaining vision after treatment with AAV-AIPL1. Beyond the effects on vision, treatment with AAV-AIPL1 resulted in life-changing benefits in vital areas of development including communication, behavior, learning, mood, psychological benefits and social integration.
Lilly will also receive worldwide exclusive access rights to MeiraGTx’s innovative gene therapy technologies for use in ophthalmology with certain targets named by Lilly, including novel intravitreal capsids developed in-house at MeiraGTx and bespoke promoters including AI-generated promoters for specific cells in the retina. MeiraGTx also grants Lilly certain rights to its proprietary riboswitch technology for use in gene editing in the eye. MeiraGTx’s riboswitch technology platform is broadly applicable to any therapeutic protein. It allows precise, titratable in-vivo production of the therapeutic protein or gene editing nuclease from a gene template controlled by oral dosing of a small molecule inducer.
Under the terms of the agreement, MeiraGTx will receive an upfront payment of $75 million and will be eligible to receive over $400 million in total milestone payments. MeiraGTx is also eligible to receive tiered royalties on licensed products.
“We are excited to be entering into this collaboration with Lilly in ophthalmology,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “MeiraGTx has been dedicated to treating patients with serious eye conditions since the company’s inception, and this collaboration with Lilly is a testament to our leadership in this field and the power of our broad toolkit of proprietary gene therapy technologies for both rare and prevalent ocular disease.”
Dr. Forbes continued, “I am very proud of the team at MeiraGTx who over the past decade has built one of the industry’s most comprehensive genetic medicine companies - from capsid screening, promoter engineering and vector optimization, to clinical development and in-house GMP manufacturing, allowing us to accelerate the development of next-generation genetic medicines from concept to clinic with exceptional speed and quality. We are particularly pleased that Lilly, a global leader in development and commercialization of innovative medicines, has chosen to partner with us in this area of high unmet need, and shares our dedication to bringing truly life changing therapies to patients with otherwise intractable conditions.”
“Ophthalmology is an emerging area of interest for Lilly,” said Andrew Adams, Lilly group vice president, Molecule Discovery. “We are excited to partner with MeiraGTx to bring transformative treatments to patients around the world suffering from eye diseases, starting with AAV-AIPL1, which has shown the unprecedented ability to restore vision in children who were born legally blind.”
About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline with four late-stage clinical programs. Each of these programs use local delivery of small doses resulting in disease modifying effects in both inherited and more common diseases, in the eye, Parkinson’s disease and radiation-induced xerostomia. MeiraGTx uses its innovative technology in optimization of capsids, promoters and novel translational control elements to develop best in class, potent, safe viral vectors. MeiraGTx’s broad pipeline is supported by end-to-end in-house manufacturing. MeiraGTx has built the most comprehensive manufacturing capabilities in the industry, with 5 facilities globally, including two that are licensed for GMP viral vector production and a GMP QC facility with clinical and commercial licensure. In addition, MeiraGTx has developed a proprietary manufacturing platform process over 9 years based on more than 20 different viral vectors with leading yield and quality aspects and commercial readiness. Uniquely, MeiraGTx has developed a novel technology for in vivo delivery of any biologic therapeutic using oral small molecules. This transformative riboswitch gene regulation technology allows precise, dose-responsive control of gene expression by oral small molecules. MeiraGTx is focusing the riboswitch platform on the regulated in vivo delivery of metabolic peptides, including GLP-1, GIP, Glucagon, Amylin, PYY and Leptin, as well as cell therapy, CAR-T for liquid and solid tumors and autoimmune diseases, and additionally PNS targets addressing long term intractable pain. MeiraGTx has developed the technology to apply genetic medicine to common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high.
About AIPL1
AAV-AIPL1 is an investigational genetic medicine for the treatment of one of the most severe forms of Leber congenital amaurosis 4 (LCA4) owing to genetic deficiency of Aryl-hydrocarbon-interacting protein-like 1 (AIPL1). It is delivered via subretinal injection to children, and through a one-time administration, AAV-AIPL1 is designed to deliver functional copies of the AIPL1 gene to cone and rod photoreceptors in the central retina, to restore vision. AAV-AIPL1 has been granted orphan drug as well as rare pediatric disease designation (RPDD) by the FDA and orphan designation by the European Commission.
About Riboswitch
MeiraGTx has developed a novel technology for in vivo delivery of any biologic therapeutic using oral small molecules. This transformative riboswitch gene regulation technology allows precise, dose-responsive control of gene expression by oral small molecules. MeiraGTx is focusing the riboswitch platform on the regulated in vivo delivery of metabolic peptides, including GLP-1, GIP, Glucagon, Amylin, PYY and Leptin, as well as cell therapy, CAR-T for liquid and solid tumors and autoimmune diseases, and additionally PNS targets addressing long term intractable pain. MeiraGTx has developed the technology to apply genetic medicine to common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high.