Lilly strikes eye-opening gene therapy deal for rare childhood blindness

Eli Lilly is acquiring exclusive rights to an experimental gene therapy for Leber congenital amaurosis-4, a rare form of childhood blindness, in a $75 million deal with MeiraGTx — plus up to $400 million in milestone payments.

Why it matters: This expands Lilly’s push into genetic medicine and eye diseases, giving it access to a promising treatment that restored vision in all trial participants.

Backstory: MeiraGTx's AAV-AIPL4 therapy showed vision restoration in 11 children during a small trial. Regulatory discussions for expedited approval are underway in the U.S. and U.K. The therapy could benefit from an FDA pilot program that fast-tracks review of treatments for pediatric blindness.

Big picture: While the target condition is ultra-rare, affecting roughly one in a million births, the deal enhances Lilly’s presence in ophthalmology and genetic medicine, following its acquisition of Adverum Biotechnologies and previous strategic investments in the space.

Zoom in: MeiraGTx has active partnerships with Sanofi (neurology/metabolism) and had previously collaborated with Johnson & Johnson. Lilly now also gains access to MeiraGTx’s gene therapy platforms, including riboswitch tech for gene regulation.