MeiraGTx Enters into Collaboration with Eli Lilly to Advance Genetic Medicines in Ophthalmology
Tuesday, November 11, 2025
MeiraGTx Holdings has entered into a strategic collaboration with Eli Lilly and Company to develop and commercialise genetic therapies targeting ophthalmic diseases.
AAV-AIPL1 is an investigational gene therapy intended for the treatment of Leber congenital amaurosis 4 (LCA4) caused by AIPL1 gene deficiency. Administered via subretinal injection, the therapy delivers functional copies of the AIPL1 gene to cone and rod photoreceptors in the central retina to restore vision. It has received Orphan Drug and Rare Paediatric Disease designations from the US FDA and Orphan Designation from the European Commission.
MeiraGTx’s riboswitch platform offers controlled, dose-responsive gene expression using orally administered small molecules. This technology can be applied across a wide range of therapeutic areas, including metabolic peptide delivery, CAR-T cell therapy, autoimmune diseases, and chronic pain management. It represents a novel approach to expanding the application of genetic medicine to both rare and prevalent conditions.
As part of the agreement, Lilly will obtain exclusive global rights to MeiraGTx’s AAV-AIPL1 programme, designed to treat Leber congenital amaurosis 4 (LCA4)—a severe inherited retinal disorder caused by mutations in the Aryl-hydrocarbon-interacting protein-like 1 (AIPL1) gene. In early clinical data, treatment with AAV-AIPL1 demonstrated significant restoration of vision in 11 children under the age of four who were born legally blind due to AIPL1 deficiency. The therapy also showed positive effects on communication, learning, behaviour, and overall quality of life.
Lilly will further gain access to MeiraGTx’s gene therapy technologies for ophthalmology, including novel intravitreal capsids, AI-generated promoters specific to retinal cells, and riboswitch technology for gene editing in the eye. The riboswitch platform enables precise, controllable production of therapeutic proteins or gene-editing nucleases, regulated through oral administration of small molecules.
Under the terms of the deal, MeiraGTx will receive an upfront payment of USD 75 million and is eligible for milestone payments exceeding USD 400 million, along with tiered royalties on future product sales.
This collaboration leverages MeiraGTx’s comprehensive expertise in genetic medicine, spanning from vector design and capsid screening to in-house GMP manufacturing and clinical development.
The partnership is expected to accelerate the advancement of next-generation gene therapies for rare and common eye diseases.
Source: meiragtx.com
