Lilly inks deal with MeiraGTx for retinal disease gene therapies

Eli Lilly has signed a broad strategic ophthalmology collaboration with MeiraGTx Holdings, in a deal that could reach over $475 million.

Per the deal, 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.

Lilly will also get worldwide exclusive rights to MeiraGTx’s 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. Additionally, Lilly will receive certain rights to MeiraGTx’s proprietary riboswitch technology for use in gene editing in the eye. The platform, which is broadly applicable to any therapeutic protein, 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 deal, 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.