MeiraGTx has granted Eli Lilly worldwide rights to its experimental AAV-AIPL1 gene therapy in a deal that could exceed $475 million. MeiraGTx will receive $75 million upfront, more than $400 million in potential milestone payments, and tiered royalties. AAV-AIPL1 is a one-time subretinal treatment for Leber congenital amaurosis type 4, intended to deliver functional AIPL1 to retinal photoreceptors in children. The agreement strengthens Lilly's push into ophthalmology and ocular gene therapies.
Eli Lilly Secures Global Rights to MeiraGTx’s AAV‑AIPL1 Gene Therapy in Deal Worth Up to $475M
MeiraGTx has granted Eli Lilly worldwide rights to its experimental AAV-AIPL1 gene therapy in a deal that could exceed $475 million. MeiraGTx will receive $75 million upfront, more than $400 million in potential milestone payments, and tiered royalties. AAV-AIPL1 is a one-time subretinal treatment for Leber congenital amaurosis type 4, intended to deliver functional AIPL1 to retinal photoreceptors in children. The agreement strengthens Lilly's push into ophthalmology and ocular gene therapies.

Eli Lilly Secures Global Rights to MeiraGTx’s AAV-AIPL1 Gene Therapy
MeiraGTx Holdings has signed an agreement with Eli Lilly that could be worth more than $475 million, granting Lilly worldwide rights to an experimental one-time gene therapy aimed at a severe, inherited form of congenital blindness.
Deal terms: MeiraGTx will receive a $75 million upfront payment and may collect more than $400 million in milestone payments, in addition to tiered royalties on any licensed products.
About the therapy: The investigational candidate, AAV-AIPL1, is being evaluated for Leber congenital amaurosis type 4 (LCA4), a severe childhood-onset retinal disorder caused by mutations in the AIPL1 gene. Administered via a single subretinal injection to children, AAV-AIPL1 is designed to deliver functional copies of AIPL1 to retinal photoreceptors to restore visual function.
Strategic context: The agreement builds on Lilly's recent push into ophthalmology and gene therapies, following its October transaction to acquire Adverum Biotechnologies (a deal valued at up to $261.7 million) for a treatment targeting wet age-related macular degeneration.
Shares of MeiraGTx rose about 12.5% to $9.55 in premarket trading after the announcement. As part of the transaction, Lilly also obtains exclusive global rights to MeiraGTx's ophthalmic gene therapy platforms and certain rights to the company's riboswitch technology for ocular gene editing.
Implications: The deal provides MeiraGTx with near-term capital to advance development while giving Lilly a stronger position in the market for advanced eye treatments and potential one-time therapies for inherited retinal diseases.
