MeiraGTx Holdings PLC has announced a broad strategic collaboration in the area of ophthalmology with Eli Lilly and Company. As part of the agreement, MeiraGTx will grant Lilly worldwide exclusive rights to its AAV-AIPL1 program for the treatment of Leber congenital amaurosis 4 (LCA4) due to genetic deficiency of Aryl-hydrocarbon-interacting protein-like 1 (AIPL1), the company said in a press release.

According to MeiraGTx, clinical data from a study of 11 children under the age of 4 were unprecedented. All 11 children, who were born legally blind as a result of mutations in the AIPL1 gene, gained vision after treatment with AAV-AIPL1. Treatment with the drug also showed benefits in vital areas of development, including communication, behavior, learning, mood, and social integration, the company said.

Lilly will also receive worldwide exclusive rights to MeiraGTx’s gene therapy technologies for use in ophthalmology with certain targets, including for novel intravitreal capsids developed in-house at MeiraGTx and bespoke promoters that include AI-generated promoters for specific cells in the retina. In addition, MeiraGTx will also grant Lilly certain rights to its proprietary riboswitch technology for use in gene editing in the eye. According to MeiraGTx, the platform is broadly applicable to any therapeutic protein, allowing 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, according to the press release. RP