Eli Lilly Inks $475M Deal to Acquire Global Rights to MeiraGTx’s AAV-AIPL1 Gene Therapy for LCA4

November 10,London and New York, vertically integrated clinical-stage gene therapy company MeiraGTx HoMeiraGTx Limited (NASDAQ: MGTX) announced a broad strategic collaboration with Eli Lilly and Company in the field of ophthalmology. Eli Lilly and Company is currently the hottest player in the CGT space.$260 Million! Eli Lilly Acquires Adverum, Gains a wAMD Gene Therapy；$1.3 Billion! Eli Lilly and Company Officially Acquires Verve (With Exclusive PPT)；$1.418 Billion! Eli Lilly and Company Reaches Licensing Agreement with Sangamo）

MeiraGTx to Grant Eli Lilly Global Exclusive Rights to Its AAV-AIPL1 Program for Treating One of the Most Severe Inherited Retinal Diseases, Leber Congenital Amaurosis 4 (LCA4), Caused by Genetic Defects in Aryl Hydrocarbon Receptor-Interacting Protein-Like 1 (AIPL1). Unprecedented clinical data from 11 children under the age of 4 born with AIPL1 mutations showed that all 11 blind children regained vision after receiving AAV-AIPL1 treatment. In addition to its impact on vision, AAV-AIPL1 treatment demonstrated significant developmental importance.The field has also brought life-changing benefits, including communication, behavior, learning, emotional, psychological benefits, and social integration.$200 Million Upfront Payment! MeiraGTx Partners with Hologen to Develop Parkinson's Gene Therapy）

Eli Lilly will also obtain the global exclusive rights to MeiraGTx's innovative gene therapy technology for ophthalmology, targeting certain indications specified by Eli Lilly. This includes novel intravitreal capsids and customized promoters developed internally by MeiraGTx, such as AI-generated retina-specific cell promoters. MeiraGTx has also granted Eli Lilly certain rights to its proprietary riboswitch technology for gene editing in the eye. MeiraGTx’s riboswitch technology platform is broadly applicable to any therapeutic protein. It enables precise, titratable in vivo production of therapeutic proteins or gene-editing nucleases from a genetic template controlled by orally administered small-molecule inducers.

According to the terms of the agreement, MeiraGTx will receive an upfront payment of $75 million and is eligible for milestone payments exceeding $400 million. MeiraGTx is also eligible for tiered royalties on licensed products.

Dr. Alexandria Forbes, President and CEO of MeiraGTx, said:"We are thrilled to collaborate with Eli Lilly in the field of ophthalmology. Since its inception, MeiraGTx has been committed to treating patients with serious eye diseases, and this collaboration with Eli Lilly underscores our leadership in this area as well as the powerful capabilities of our broad proprietary gene therapy technology toolkit for both rare and prevalent eye conditions. I am extremely proud of the MeiraGTx team, who over the past decade have built one of the most comprehensive gene therapy companies in the industry — from capsid screening, promoter engineering, and vector optimization to clinical development and internal GMP manufacturing — enabling us to accelerate the development of next-generation gene therapies from concept to clinic with extraordinary speed and quality. We are particularly pleased that Eli Lilly, a global leader in innovative drug development and commercialization, has chosen to partner with us in this underserved area, and shares our commitment to bringing truly life-changing therapies to patients suffering from other challenging diseases."

Andrew Adams, Vice President of Molecular Discovery at Eli Lilly and Company, stated:"Ophthalmology is an emerging field of interest for Eli Lilly. We are excited to collaborate with MeiraGTx to bring transformative treatments to patients suffering from eye diseases around the world, starting with AAV-AIPL1, which has already demonstrated an unprecedented ability to restore vision in children who are legally blind from birth."

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