MeiraGTx Holdings plc, a clinical stage genetic medicines company, entered a broad strategic collaboration in the area of ophthalmology with Eli Lilly and Co.

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 demonstrated that all 11 blind children gained vision after treatment with AAV-AIPL1.

Lilly will also receive worldwide exclusive access rights to MeiraGTx’s gene therapy technologies for use in ophthalmology with certain targets named by Lilly. 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 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.”

“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.”