Eli Lilly Deepens Gene Therapy Commitment with MeiraGTx Collaboration, Expanding Ophthalmology Focus
Eli Lilly is substantially expanding its gene therapy portfolio, most recently through a strategic collaboration with MeiraGTx focused on inherited retinal diseases.This partnership underscores Lilly’s growing commitment to genomic medicine and its ambition to deliver perhaps curative treatments for debilitating conditions. Here’s a detailed look at teh deal and how it fits into Lilly’s broader strategy.
The MeiraGTx Deal: A Focus on Restoring Vision
Lilly has secured exclusive rights to develop and commercialize MeiraGTx’s AAV-AIPL1 gene therapy, a promising candidate for treating AIPL1-related Leber congenital amaurosis (LCA).LCA is a rare, inherited form of blindness that typically manifests in infancy. Early data suggests AAV-AIPL1 has demonstrated the remarkable ability to restore vision in children born legally blind.
The financial terms are substantial. MeiraGTx will receive an upfront payment of $300 million, with the potential for up to $700 million in additional milestone payments tied to regulatory approvals and commercial success. Furthermore,Lilly will pay royalties on future sales of the commercialized product.
But the collaboration extends beyond AAV-AIPL1. Lilly also gains rights to:
* Expand into Other Retinal Diseases: Lilly now has the prospect to develop and commercialize additional preclinical gene therapies from MeiraGTx targeting other inherited retinal dystrophies – the specific diseases remain undisclosed.
* Proprietary Delivery Technology: Lilly gains exclusive licenses to meiragtx’s advanced gene therapy delivery components:
* Up to five ophthalmologic targets can be selected for use with MeiraGTx’s intravitreal capsids (the protein shells that deliver genetic material).
* up to five targets can utilize MeiraGTx’s pan-retinal or rod-specific promoters.
* Gene Editing Capabilities: Access to MeiraGTx’s riboswitch technology, enabling in vivo production of therapeutic proteins controlled by an oral small molecule, opens new avenues for gene editing in the eye.
Lilly’s Rapid Ascent in Gene Therapy
This deal isn’t an isolated event. Over the past few years, Lilly has been aggressively building a leading position in gene therapy through a series of strategic acquisitions and partnerships. You’re witnessing a clear and intentional strategy to diversify beyond customary pharmaceuticals.
Here’s a timeline of Lilly’s key moves:
* 2021: Prevail Therapeutics Acquisition: Lilly acquired Prevail Therapeutics, focusing on gene therapies for neurodegenerative and rare disorders. Prevail afterward formed an R&D alliance with Capsida Biotherapeutics to expand its CNS gene therapy pipeline.
* 2022: Akouos Buyout: Lilly purchased Akouos, a company developing a gene therapy for a rare, inherited form of hearing loss.
* Early 2023: Sangamo Therapeutics Capsid License: Lilly secured rights to a capsid from Sangamo Therapeutics for delivering genomic medicines to the central nervous system.
* Late 2023: Adverum Biotechnologies Acquisition: Lilly acquired Adverum Biotechnologies, adding a Phase 3 gene therapy program for wet age-related macular degeneration to its portfolio.
* April 2024: MeiraGTx Collaboration: The latest partnership, focusing on inherited retinal diseases and expanding Lilly’s ophthalmology presence.
Why Ophthalmology?
Andrew Adams, Lilly’s group vice president of molecule discovery, highlighted ophthalmology as an “emerging area of interest” for the company. This makes sense. Gene therapy holds immense promise for treating genetic forms of blindness and other eye diseases where delivering the therapeutic directly to the eye is relatively straightforward.
What This Means for Patients
lilly’s investment in gene therapy, and specifically its focus on inherited retinal diseases, offers real hope for patients with limited or no treatment options. The potential to restore vision in children born with LCA, as demonstrated by AAV-AIPL1, is a game-changer.
As Lilly continues to integrate these technologies and advance its pipeline, you can expect to see further innovation and, ultimately, more life-
