Eli Lilly and Company entered into a deal with MeiraGTx in which it will acquire worldwide exclusive rights to MeiraGTx's AAV-AIPL1, a gene therapy program for the treatment of Leber congenital amaurosis type 4 (LCA4).  

MeiraGTx will receive an upfront payment of $75 million and will be eligible for more than $400 million in milestone payments, along with tiered royalties on any licensed products that reach the market.

The deal with MeiraGTx comes just 2 weeks after Lilly entered a definitive agreement to acquire Adverum Biotechnologies, including its lead gene therapy product candidate, Ixo-vec, for the treatment of wet age-related macular degeneration (AMD).

According to MeiraGTx, clinical data from a trial involving 11 children under the age of 4, all born legally blind due to AIPL1 mutations, demonstrated promising outcomes. Every treated child gained measurable vision following treatment with AAV-AIPL1. Beyond restoring sight, the therapy also produced transformative improvements in communication, learning, mood, and social integration.

In addition to AAV-AIPL1, Lilly will obtain exclusive access rights to MeiraGTx’s gene therapy platform technologies for ophthalmology, covering specific targets designated by Lilly. This includes MeiraGTx’s novel intravitreal capsids and AI-generated cell-specific promoters, engineered to enhance gene delivery and expression in the retina. The collaboration also grants Lilly access to MeiraGTx’s proprietary riboswitch technology, enabling precise, titratable control of gene expression or gene editing in the eye. This platform allows in-vivo modulation of therapeutic protein production through an orally administered small molecule, offering unique control and safety advantages in gene therapy.

"This collaboration underscores our leadership in the field and the power of our proprietary gene therapy technologies for both rare and prevalent ocular diseases," said Alexandria Forbes, PhD, president and chief executive officer of MeiraGTx. “I am incredibly proud of the MeiraGTx team, who have built one of the industry’s most comprehensive genetic medicine platforms—from vector optimization and promoter engineering to in-house GMP manufacturing. Lilly’s partnership reflects a shared commitment to delivering transformative treatments for patients with otherwise intractable conditions.”