MeiraGTx is cashing in on a gene therapy that restored vision to 11 children who were born legally blind, licensing the program to Eli Lilly in a deal potentially worth more than $475 million.
The Indiana drugmaker will gain exclusive global rights to the AAV-AIPL1 program for the treatment of Leber congenital amaurosis type 4 (LCA4), a severe retinal disease present at birth that can be caused by genetic mutations that result in low levels of Aryl-hydrocarbon-interacting protein-like 1 (AIPL1). MeiraGTx announced the deal in a Nov. 10 release.
The deal also gives Lilly access rights to other ophthalmology gene therapy technologies developed by MeiraGTx, like novel capsids for drug delivery and AI-designed promoters targeting certain cells in the retina, MeiraGTx said in the release, as well as some access to the company’s riboswitch platform, a method for generating therapeutic proteins in vivo.
New York-based MeiraGTx will net $75 million upfront, with more than $400 million in further payments possible if certain milestones are met, along with royalties on sales of any resulting products.
“Ophthalmology is an emerging area of interest for Lilly,” Andrew Adams, Ph.D., Lilly’s group vice president of molecule discovery, said in the release. “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.”
MeiraGTx first teased that result in February, publishing data from four of the 11 children that showed the gene therapy had improved visual acuity in treated eyes. In its Nov. 10 release, the biotech said that all 11 children gained vision as a result of the treatment.
In the company’s second-quarter earnings release on Aug. 14, MeiraGTx said it was on track to submit the LCA4 gene therapy for approval in the U.K., with a similar submission in the U.S. planned for the fourth quarter of 2025.
Not long after releasing the vision-restoring data that caught Lilly’s eye, MeiraGTx announced it had found a partner for a different gene therapy. In March, Hologen AI committed up to $430 million to help take MeiraGTx’s Parkinson’s disease gene therapy through phase 3 and commercialization.
Lilly made another eye disease gene therapy move less than a month ago, swooping in to snatch up Adverum Biotechnologies just as the biotech’s funds were running dry. Adverum’s star candidate is ixo-vec, a gene therapy for wet age-related macular degeneration that entered a phase 3 trial in March and which Adverum has hailed as a one-and-done treatment that could “preserve sight for life.”