India Pharma Outlook Team | Tuesday, 11 November 2025
Eli Lilly and Company has signed an agreement with MeiraGTx Holdings plc that grants Lilly global exclusive rights to MeiraGTx’s investigational gene therapy for a severe inherited retinal condition.
The centerpiece of the deal is the programme AAV?AIPL1, designed to treat Leber congenital amaurosis 4 (LCA4) — a disorder that causes profound vision loss from birth.
The gene therapy deal opens a new chapter in the pharma industry’s push into rare eye disorders. Under the terms, MeiraGTx receives an upfront payment of?US?$75?million, with the potential to earn over?$400?million in milestone payments.
On top of that, tiered royalties on commercial sales are included. These financials reflect both the promise and risk inherent in gene therapy investments.
Also Read: Importance of Investing in R&D and Quality for Competitive Advantage in Eye Care
From an industry perspective, the gene therapy deal confirms Lilly’s clear intent to strengthen its foothold in both rare ophthalmic diseases and advanced modalities. The firm already made a related move in October when it acquired Adverum Biotechnologies in a deal worth up to?$261.7?million targeting age?related macular degeneration.
Clinical results to date for AAV?AIPL1 show remarkable promise: in a small trial of children born legally blind due to AIPL1 mutations, all treated participants demonstrated improvements in vision after a single sub?retinal injection. While the rare nature of LCA4 limits the market size, the one?time treatment concept and transformative potential make this deal significant for the gene therapy sector.
Overall, this collaboration signals increasing confidence among big pharma players in gene therapies for rare ophthalmic disorders — moving from small?scale lab concepts toward major commercial plays. With Lilly leveraging its global development and commercialization expertise, the partnership could accelerate access to this next?generation treatment if clinical and regulatory milestones are met.
