Eli Lilly has agreed to a $475m licensing deal with MeiraGTx, marking the second eye disease gene therapy bet by the drugmaker in as many months.
Eli Lilly will pay UK-based MeiraGTx $75m upfront for global exclusive rights to AAV-AIPL1, a gene therapy in development for a rare form of inherited blindness, along with access to further gene therapy technologies developed by the biotech.
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The collaboration also includes more than $400m in milestone payments, as well as royalties on any drugs approved in the future.
MeiraGTx’s AAV-AIPL1 is in development to treat Leber congenital amaurosis type 4 (LCA4), a severe inherited retinal disease that is caused by genetic deficiency of aryl-hydrocarbon-interacting protein-like 1 (AIPL1).
AIPL1 is a protein critical for the functioning of the eye’s light-sensing pathway.
AAV-AIPL1 has already produced positive results in a non-randomised study conducted in the UK, with results published in The Lancet in February 2025. Data from four of 11 children enrolled in the study, who had severe retinal dystrophy due to AIPL1 mutation, demonstrated that the therapy improved visual acuity in treated eyes.
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By GlobalDataIn its press release announcing the Lilly collaboration, MeiraGTx said that AAV-AIPL1 led to vision gains in all 11 treated children, along with benefits in other areas of paediatric development.
Andrew Adams, Lilly’s group vice-president of molecule discovery, said: “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, which has shown the unprecedented ability to restore vision in children who were born legally blind.”
In a quarterly report published in June 2025, MeiraGTx said that it is “preparing the submission of an marketing authorisation application in the UK and a biologics license application in the US for AAV-AIPL1”. This was based on discussions with the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and US Food and Drug Administration (FDA) and the results from the 11 patients.
Other ophthalmology assets included in the deal are intravitreal capsids developed in-house at MeiraGTx and AI-generated promoters for specific cells in the retina. The biotech has also opened the door to Lilly for its riboswitch technology, a platform that allows in-vivo production of a therapeutic protein or gene editing nuclease.
Adams’ statement that ophthalmology represents an “emerging area of interest” for Lilly is supported by the money the company has recently spent in the area. last month, Lilly outlaid $261.7m to acquire Adverum Biotechnologies, a company developing an intravitreal gene therapy for the treatment of wet age-related macular degeneration (AMD). Combined with the MeiraGTx licensing deal, this means Lilly has spent more than half a billion dollars on gene therapies specifically in development for eye diseases.
This is not the only gene therapy area Lilly has targeted in 2025. In April, the big pharma company signed a licensing deal worth up to $1.4bn for Sangamo Therapeutics’ neurology-targeting gene therapy. In addition, Lilly acquired RNA-based gene therapy developer Rznomics in May 2025, and gene editing partner Verve Therapeutics for $1.3bn a month later.
Lilly’s R&D push with the modality goes against the grain of big pharma generally opting to retreat from the cell and gene therapy sector.
Earlier this month, Galapagos wound down its cell and gene therapy division after failing to sell the unit. Japanese pharma Takeda also abandoned its cell therapy research, pivoting instead towards small molecules, biologics and antibody-drug conjugates (ADCs).
In addition, Gilead Sciences’ Kite Pharma terminated its cell therapy collaboration with Shoreline in September 2025, ending a research partnership valued at $2.3bn.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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