Eli Lilly has struck a global research and licensing collaboration with Ascidian Therapeutics focused on RNA exon editing therapeutics for inherited kidney diseases, a deal that could be worth up to $1.9 billion. Ascidian’s platform is designed to correct disease-causing exons at the RNA level using endogenous splicing machinery rather than permanent DNA editing. Under the agreement, Ascidian will lead discovery and early preclinical work while Lilly takes responsibility for later-stage preclinical development, clinical trials, manufacturing, and commercialization. Lilly receives exclusive rights for undisclosed monogenic kidney targets, with options to expand into additional targets. The partnership adds a new modality to Lilly’s genetic medicine portfolio and targets a large unmet need: more than 60 genetically driven kidney diseases with few effective options beyond symptom management, transplantation, or dialysis. Ascidian positions its approach as enabling small “kilobase-scale” RNA payloads that can fit in delivery formats such as AAV. Separately, the expanded deal reinforces industry momentum around RNA-based gene repair strategies that aim to reduce risks associated with genome editing while maintaining the ability to address mutation patterns spread across multiple exons.
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