Lilly Secures AAV-AIPL1 Gene Therapy Rights from MeiraGTx in $475M Deal

LONDON and NEW YORK, Nov. 10, 2025 (GLOBE NEWSWIRE) –Eli Lilly and Company today announced a major licensing agreement with UK-based MeiraGTx, securing global exclusive rights to AAV-AIPL1, an investigational AAV gene therapy designed to treat a rare form of inherited blindness. The deal, valued at up to $475 million, includes a $75 million upfront payment to MeiraGTx, over $400 million in potential milestone payments, and future royalties.

This agreement marks Eli Lilly’s second significant investment in AAV gene therapy for eye diseases in as many months, signaling ophthalmology as a critical emerging area for the pharmaceutical giant.

AAV-AIPL1: Targeting LCA4

AAV-AIPL1 is an AAV-delivered gene therapy in development for Leber congenital amaurosis type 4 (LCA4), a severe inherited retinal disease caused by a genetic deficiency of the AIPL1 protein, which is critical for the eye’s light-sensing pathway.

The AAV gene therapy has already shown positive results in a non-randomized study conducted in the UK, with findings published in The Lancet in February 2025. Data from 11 treated children showed AAV-AIPL1 led to vision gains in all participants, along with benefits in other areas of pediatric development. For children born legally blind, the therapy has demonstrated the “unprecedented ability to restore vision.”

MeiraGTx is currently preparing the submission of a marketing authorization application in the UK and a biologics license application in the US for the AAV treatment, following encouraging discussions with the MHRA and FDA based on the 11 patient results.

Lilly’s Growing AAV and Gene Therapy Portfolio

The license for AAV-AIPL1 provides Lilly with global rights to the investigational treatment and access to MeiraGTx’s broader gene therapy technologies. The collaboration also includes rights to intravitreal capsids developed by MeiraGTx and AI-generated promoters for specific cells in the retina.

Andrew Adams, Lilly’s group vice-president of molecule discovery, confirmed the strategic focus: “Ophthalmology is an emerging area of interest for Lilly. We are excited to partner with MeiraGTx to bring transformative treatments to patients around the world suffering from eye diseases, starting with AAV-AIPL1.”

Lilly’s commitment to AAV and gene therapy has seen rapid escalation throughout 2025. This deal follows the acquisition of AAV developer Adverum Biotechnologies last month for $261.7 million, alongside significant investments in neurology and other genetic modalities through deals with Sangamo Therapeutics, Rznomics, and Verve Therapeutics. This aggressive R&D push in cell and gene therapy bucks the recent trend of some big pharma companies, such as Galapagos and Takeda, scaling back or abandoning their respective gene therapy divisions.