MeiraGTx Partners with Eli Lilly in $475M Gene Therapy Deal to Restore Vision in Children

MeiraGTx Holdings plc has announced a broad strategic collaboration with Eli Lilly and Company focused on advancing gene therapies for ophthalmic diseases, including a groundbreaking treatment for Leber congenital amaurosis 4 (LCA4) — a rare inherited retinal disorder causing blindness in children.

Under the agreement, Lilly gains exclusive global rights to MeiraGTx’s AAV-AIPL1 program, which has demonstrated remarkable clinical results: all 11 treated children, previously born legally blind, gained vision following therapy. Beyond visual improvement, the treatment also delivered significant benefits in learning, behavior, and social development.

The partnership also grants Lilly access to MeiraGTx’s gene therapy technologies for ophthalmology, including next-generation intravitreal capsids, AI-generated retina-specific promoters, and the company’s proprietary riboswitch platform, which enables controlled, dose-dependent gene expression.

MeiraGTx will receive $75 million upfront and up to $400 million in milestone payments, plus tiered royalties on product sales.

“We are thrilled to collaborate with Lilly, a global leader in innovative medicine,” said Dr. Alexandria Forbes, President and CEO of MeiraGTx. “This partnership validates our leadership in ocular gene therapy and our commitment to transforming lives of patients with severe eye conditions.”

Lilly’s Andrew Adams, Group Vice President of Molecule Discovery, added, “AAV-AIPL1 has shown the unprecedented ability to restore vision in children born blind. We are excited to advance this transformative therapy worldwide.”