MeiraGTx Enters into Strategic Collaboration with Lilly for Genetic Medicines for Eye Diseases

Rare Daily Staff

MeiraGTx said it entered a broad strategic collaboration in ophthalmology with Eli Lilly to develop and commercialize genetic medicines to treat eye diseases. The agreement includes $75 million in upfront payments and up to more than $400 million in milestone payments.

MeiraGTx is also eligible for tiered royalties on licensed products.

Under the agreement, MeiraGTx will grant Lilly worldwide exclusive rights to its AAV-AIPL1 program for the treatment of Leber congenital amaurosis 4, a severe inherited retinal disease.

AAV-AIPL1 is an experimental genetic medicine designed to treat one of the most severe forms of Leber congenital amaurosis 4 (LCA4) caused by a genetic deficiency of aryl-hydrocarbon-interacting protein-like 1 (AIPL1). Delivered via subretinal injection to children through a one-time administration, AAV-AIPL1 is designed to provide functional copies of the AIPL1 gene to cone and rod photoreceptors in the central retina to restore vision. AAV-AIPL1 has been granted Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration, as well as Orphan Designation from the European Commission.

Clinical data from 11 children under the age of 4 who were born legally blind due to mutations in the AIPL1 gene were unprecedented, with all 11 children gaining vision after treatment with AAV-AIPL1.

Beyond restoring vision, treatment with AAV-AIPL1 resulted in life-changing benefits in vital areas of development, including communication, behavior, learning, mood, psychological health, and social integration.

As part of the agreement, Lilly will receive exclusive worldwide rights to MeiraGTx’s gene therapy technologies for use in ophthalmology, with certain targets named by Lilly. These include novel intravitreal capsids developed in-house at MeiraGTx and bespoke promoters, including AI-generated promoters designed for specific cells in the retina.

MeiraGTx also granted Lilly certain rights to its proprietary riboswitch technology for use in gene editing in the eye. The company’s riboswitch technology platform is applicable to any therapeutic protein and 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.

“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,” said Alexandria Forbes, president and chief executive officer of MeiraGTx. “We are particularly pleased that Lilly, a global leader in the development and commercialization of innovative medicines, has chosen to partner with us in this area of high unmet need.”

Photo: Alexandria Forbes, president and chief executive officer of MeiraGTx