Eli Lilly and Ascidian Therapeutics signed a global research and licensing collaboration for RNA exon editing therapies targeting inherited monogenic kidney diseases. Lilly will pay up to $1.9 billion across upfront fees, development and commercial milestones, and royalties, with Ascidian leading discovery and early preclinical work before Lilly takes over later-stage development, clinical testing, manufacturing, and commercialization. The technology is designed to correct disease-causing mutations at the RNA level by using the cell’s splicing machinery to replace targeted exons via trans-splicing, rather than editing DNA. Ascidian will retain freedom to pursue other kidney disease programs outside the collaboration. The deal expands Lilly’s push into genetic medicines and highlights growing interest in RNA-based approaches that may broaden addressable mutation sets for hard-to-edit kidney targets.
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