MeiraGTx Holdings plc and Eli Lilly & Co. have entered into a strategic collaboration that grants Lilly worldwide exclusive rights to MeiraGTx’s AAV‑AIPL1 gene‑therapy program for Leber congenital amaurosis type 4 (LCA4) and access to several of the company’s proprietary platforms, including intravitreal capsids, AI‑generated retinal promoters, and a riboswitch system for precise control of therapeutic protein expression.
Under the agreement, MeiraGTx will receive a $75 million upfront payment and is eligible for more than $400 million in milestone payments, plus tiered royalties on any licensed products. The upfront cash is a substantial boost for a company that reported a negative free‑cash‑flow of roughly $134 million over the last twelve months, providing liquidity to fund ongoing clinical development and future pipeline projects.
The AAV‑AIPL1 therapy has already shown remarkable clinical results, with 11 children under four years old regaining functional vision after a single intravitreal injection. The program has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration and an orphan designation from the European Commission, underscoring its potential to address a severe, untreatable inherited retinal disease.
For MeiraGTx, the partnership validates its gene‑therapy platform and expands its commercialization reach through Lilly’s global distribution network. For Lilly, the deal marks a significant entry into the ophthalmology sector and adds a high‑potential, early‑stage therapy to its gene‑therapy pipeline, complementing recent acquisitions such as Adverum Biotechnologies. The collaboration is expected to accelerate development timelines and bring the therapy closer to regulatory approval.
Alexandria Forbes, CEO of MeiraGTx, said the collaboration “demonstrates the strength of our technology portfolio and provides the capital and expertise needed to bring a life‑changing therapy to patients worldwide.” Andrew Adams, Lilly’s group vice‑president of Molecule Discovery, added that the partnership “expands Lilly’s ophthalmology portfolio and leverages our global reach to deliver transformative treatments to patients with rare eye diseases.” Analysts noted that the deal’s financial terms and the clinical promise of AAV‑AIPL1 are likely to support a positive outlook for both companies.
With the upfront payment and potential milestone upside, MeiraGTx now has a stronger balance sheet to sustain its clinical program and pursue additional gene‑therapy candidates. Lilly’s acquisition of exclusive rights to the LCA4 program and its riboswitch technology positions the company to capitalize on the growing gene‑therapy market and to diversify its portfolio beyond its existing therapeutic areas.
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