MeiraGTX, a clinical-stage genetic medicines company, announced a strategic collaboration deal with Eli Lilly and Company on November 10th, 2025. Lilly will pay up to $475 million, plus tiered royalties, for the worldwide exclusive rights to MeiraGTX’s AAV-AIPL1 program. AAV-AIPL1 is intended as a treatment for Leber congenital amaurosis 4, a severe inherited retinopathy that results in severe vision loss at an early age.
Delivered via a one-time, subretinal injection to children, AAV-AIPL1 is designed to deliver functional copies of the AIPL1 gene to cone and rod photoreceptors in the central retina. According to the release, clinical data from a study on eleven children under the age of 4 who were born legally blind due to mutations in the AIPL1 gene showed that all subjects gained vision after receiving treatment with AAV-AIPL1. The treatment has been granted orphan drug and rare pediatric disease designation by FDA, as well as an orphan designation from the European Commission.
In addition to this indication, Lilly received worldwide access rights to MeiraGTX’s gene therapy technologies for use in investigating other novel targets in the field of ophthalmology. This consists of novel intravitreal capsids developed at MeiraGTx and bespoke promoters, including AI-generated promoters for specific cells in the retina. The deal also grants Lilly rights to MeiraGTX’s riboswitch technology platform for use in gene editing in the eye. Riboswitch is a gene regulation technology designed to provide precise, dose-responsive control of gene expression through small oral molecules.
“We are excited to be entering into this collaboration with Lilly in ophthalmology,” said Alexandria Forbes, Ph.D., president and CEO, MeiraGTx, in the release. “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.”
“Ophthalmology is an emerging area of interest for Lilly,” said Andrew Adams, Vice President, Molecule Discovery, Eli Lilly, in the release. “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.”
