Eli Lilly entered a global research and licensing collaboration with Ascidian Therapeutics worth up to $1.9 billion to pursue RNA exon editor therapies for inherited kidney diseases. Ascidian’s technology uses RNA exon editing—via engineered enzymes and cellular splicing machinery—to replace faulty exon segments at the RNA level without permanent DNA alteration. Under the agreement, Ascidian will lead discovery and early preclinical activities, while Lilly will handle later-stage preclinical development, clinical trials, manufacturing, and commercialization. The initial focus is on undisclosed monogenic kidney targets, with Lilly retaining exclusive rights for those agreed targets and an option to add additional ones. The deal adds another platform-driven bet to Lilly’s genetic medicines pipeline and positions RNA editing as an approach intended to expand delivery options beyond permanent genome editing and some constraints of DNA-targeted systems.
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