Eli Lilly Deepens Gene Therapy Bet With $475 Million MeiraGTx Deal

Eli Lilly and Company (NYSE: LLY) is doubling down on ophthalmic gene therapy, announcing a $475 million licensing deal with UK-based MeiraGTx Holdings plc to develop and commercialize a groundbreaking treatment for a rare inherited form of blindness. Under the agreement, Lilly will pay $75 million upfront for exclusive global rights to MeiraGTx’s AAV-AIPL1 therapy and access to its broader gene therapy technology platform. The deal also includes over $400 million in potential milestone payments and future royalties on approved products.

AAV-AIPL1 targets Leber congenital amaurosis type 4 (LCA4)... a severe retinal disorder caused by mutations in the AIPL1 gene, which is essential to the eye’s light-sensing process. In February 2025, The Lancet published data from a UK non-randomized study showing meaningful gains in visual acuity in all 11 treated children, several of whom were born legally blind.

“Ophthalmology is an emerging area of interest for Lilly,”
said Andrew Adams, Lilly’s group vice president of molecule discovery.
“We are excited to partner with MeiraGTx to bring transformative treatments to patients suffering from eye diseases, starting with AAV-AIPL1, which has shown the unprecedented ability to restore vision in children who were born legally blind.”

MeiraGTx said it plans to submit regulatory filings in both the U.K. and U.S. based on its discussions with the MHRA and FDA, supported by data from the initial trial. Beyond AAV-AIPL1, the partnership gives Lilly access to MeiraGTx’s intravitreal capsids, AI-generated retinal promoters, and riboswitch technology, which allows for targeted gene expression within the eye.

The MeiraGTx collaboration follows Lilly’s $261.7 million acquisition of Adverum Biotechnologies last month, its second major ophthalmology investment in 2025. Together, the two transactions push Lilly’s gene therapy outlay for eye diseases beyond half a billion dollars this year.

Lilly’s rapid expansion in the modality also includes licensing and acquisition deals in other therapeutic areas:

• A $1.4 billion partnership with Sangamo Therapeutics in April for neurology-targeted gene therapy.
   
• The acquisition of Rznomics in May 2025 to bolster RNA-based delivery capabilities.
   
• The purchase of Verve Therapeutics for $1.3 billion in June, expanding its gene editing footprint.

These moves run counter to the broader industry retrenchment in cell and gene therapy. Firms like Galapagos, Takeda, and Gilead’s Kite Pharma have all scaled back or exited their programs this year following high costs and limited returns. While many pharmaceutical peers have pivoted toward small molecules, biologics, and antibody-drug conjugates, Lilly’s approach signals long-term confidence in gene therapy’s commercial viability. Analysts say the MeiraGTx deal underscores the company’s intent to own a leadership position in next-generation ophthalmics… one of the few remaining areas of gene therapy to show consistent clinical success.

About Eli Lilly and Company

Eli Lilly and Company (NYSE: LLY) is a global pharmaceutical company headquartered in Indianapolis, Indiana. Founded in 1876, Lilly discovers, develops, and markets medicines in areas including diabetes, obesity, neuroscience, oncology, and immunology. The company is expanding into gene and RNA-based therapies through strategic acquisitions and partnerships aimed at addressing serious diseases with unmet medical needs.

About MeiraGTx Holdings plc

MeiraGTx Holdings plc (NASDAQ: MGTX) is a clinical-stage gene therapy company headquartered in London, U.K., with operations in the U.S. and Ireland. Founded in 2015, MeiraGTx focuses on developing gene therapies for serious diseases, including inherited retinal disorders, neurodegenerative conditions, and gland-related disorders. The company integrates proprietary vector engineering, AI-based promoter design, and advanced manufacturing to support its growing pipeline.

At the time of publishing, Stocks.News does not hold positions in companies mentioned in the article.