AAV-AIPL1

New York-based MeiraGTx will net $75 million upfront, with more than $400 million in further payments possible if certain milestones are met.\n 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.”

Eli Lilly has struck a deal worth potentially more than $475 million with MeiraGTx to license a gene therapy that allowed children born blind to gain sight. The agreement grants Lilly exclusive worldwide rights to AAV-AIPL1, which targets Leber congenital amaurosis 4, a rare inherited retinal disorder caused by mutations in the AIPL1 gene.Andrew Adams, Lilly's head of molecule discovery, said AAV-AIPL1's results so far point to "unprecedented ability to restore vision," adding that ophthalmology is "an emerging area of interest" for the company. Lilly recently acquired Adverum Biotechnologies for its wet age-related macular degeneration gene therapy candidate.As part of Monday's deal, MeiraGTx will receive $75 million upfront as part of the deal, and is eligible for more than $400 million in milestone payments, plus tiered royalties on licensed products.According to MeiraGTx, all 11 children under age four born legally blind as a result of mutations in the AIPL1 gene and who received the treatment regained vision. Data from four patients with AIPL1-associated severe retinal dystrophy were published in The Lancet earlier this year.Administered via subretinal injection, AAV-AIPL1 is designed to deliver functional copies of the AIPL1 gene to cone and rod photoreceptors in the central retina. Beyond its effects on vision, MeiraGTx said the one-time therapy yielded improvements in important developmental areas as well, including communication, behaviour, learning, mood, psychological benefits and social integration.The alliance also gives Lilly exclusive access rights to MeiraGTx's broader genetic medicine toolkit for use in ophthalmology, with certain targets named by Lilly, including intravitreal capsids and AI-generated promoters for specific cells in the retina. Additionally, Lilly gains certain rights to MeiraGTx's proprietary "riboswitch" technology, a platform that allows precise, titratable in vivo production of a therapeutic protein or gene editing nuclease from a gene template controlled by oral dosing of a small molecule inducer, according to the companies.The deal comes as the gene therapy space has been making somewhat of a comeback. After a tough 2024, when investors questioned whether million-dollar gene therapies could really succeed in the market, some companies, including MeiraGTx, have gotten a boost since the FDA's signalling of extra flexibility for rare diseases (see – Vital Signs: A rare rebound for gene therapy).