Eli Lilly Strikes Multiple Deals for Ultra-Rare Eye Disease Gene Therapy and RNAi Partnerships

Eli Lilly secured exclusive rights to MeiraGTx’s experimental gene therapy AAV-AIPL4 for Leber congenital amaurosis-4, an ultra-rare inherited blinding eye disorder, in a deal including $75 million upfront and up to $400 million in milestone payments, plus royalties1 3 5 6.

The therapy AAV-AIPL4 demonstrated restored visual function in all 11 children enrolled in a small trial; Lilly and MeiraGTx are seeking expedited approval in the US and UK1.

The MeiraGTx deal also includes access to gene therapy tools and riboswitch gene-regulation technology for broader eye disease drug development1.

Separately, Eli Lilly entered a collaboration with Genetic Leap, a biotech using AI models to discover RNA-targeted drugs, in a deal worth up to $409 million in milestone payments plus tiered royalties; the focus is on generating oligonucleotide drug candidates against selected targets in key strategic areas like neurodegeneration, diabetes, and obesity2.

Eli Lilly signed a $1.2 billion deal with SanegeneBio to discover and optimize RNAi medicines for cardiometabolic diseases using Sanegene’s LEAD platform, including upfront payments and equity investment4 7.

Lilly’s recent moves are part of a broader strategy; in August 2024, Lilly opened a $700 million nucleic acid R&D center in Boston to expand its DNA and RNA medicines research capabilities2 4.