Lilly Acquires MeiraGTx Gene Therapy for LCA4

Eli Lilly and Company has entered into a licensing agreement with MeiraGTx to acquire worldwide exclusive rights to AAV-AIPL1, an investigational gene therapy for Leber congenital amaurosis type 4 (LCA4), a rare, inherited retinal disorder that causes severe visual impairment from birth.

As part of the agreement:

• MeiraGTx will receive a $75 million upfront payment

• The company is eligible for over $400 million in milestone payments

• MeiraGTx will also receive tiered royalties on future product sales

Back-to-Back Ophthalmic Acquisitions by Eli Lilly

This marks Lilly’s second major ophthalmic acquisition in just two weeks, following a definitive agreement to acquire Adverum Biotechnologies. That deal included Adverum’s lead gene therapy candidate Ixo-vec, currently under development for wet age-related macular degeneration (AMD).

Together, these strategic moves reinforce Lilly’s expanding presence in the ophthalmic gene therapy landscape, targeting both rare and prevalent retinal diseases.

Clinical Data: Vision Restoration in Blind Children

According to MeiraGTx, early clinical results from a study involving 11 children under the age of 4, all born legally blind due to AIPL1 mutations, showed highly encouraging outcomes:

• All treated patients experienced measurable improvements in vision

• Additional benefits included improved communication, learning abilities, mood, and social integration

These results position AAV-AIPL1 as a potential transformative therapy for one of the most severe inherited retinal dystrophies.

Access to MeiraGTx’s Gene Therapy Platform Technologies

In addition to acquiring rights to AAV-AIPL1, Lilly will gain exclusive access to MeiraGTx’s ophthalmology gene therapy platform technologies, including:

• Novel intravitreal capsids for enhanced retinal gene delivery

• AI-generated, cell-specific promoters to improve gene expression targeting

• Proprietary riboswitch technology enabling titratable in vivo gene expression control

This riboswitch system allows precise modulation of therapeutic protein production through an orally administered small molecule, adding an additional layer of safety and control to gene therapies for the eye.

Executive Commentary

Alexandria Forbes, PhD, President and CEO of MeiraGTx, expressed enthusiasm for the collaboration:

“We are excited to be entering into this collaboration with Lilly in ophthalmology. MeiraGTx has been dedicated to treating patients with serious eye conditions since the company’s inception, and this collaboration with Lilly is a testament to our leadership in this field and the power of our broad toolkit of proprietary gene therapy technologies for both rare and prevalent ocular disease.”