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Lilly Wins Exclusive License to Ascidian RNA Exon-Editing Platform for Kidney Diseases

The agreement hands later-stage development and commercialization to Lilly while expanding its genetic-medicine pipeline and funding Ascidian’s early research efforts.

Overview

  • Ascidian Therapeutics signed a licensing deal with Eli Lilly that grants Lilly exclusive rights to use Ascidian’s RNA exon-editing technology for certain undisclosed rare inherited kidney-disease targets and is in effect as of June 3, 2026.
  • The deal is worth up to $1.9 billion to Ascidian and includes an undisclosed upfront payment plus development, regulatory and sales milestone payments and tiered royalties on future product sales.
  • Under the agreement Ascidian will lead discovery work and some preclinical studies while Lilly will take responsibility for later preclinical work, clinical development, manufacturing and global commercialization.
  • Ascidian’s RNA exon-editing platform is designed to alter RNA transcripts to correct gene expression without permanently changing a patient’s DNA, an approach the company says may avoid risks tied to permanent DNA edits.
  • The transaction follows Ascidian’s 2024 license to Roche for neurological targets and fits Lilly’s recent string of moves to build a genetic-medicine business, a shift that could speed development of new therapies for people with rare inherited kidney disorders and reshape specialty drug portfolios.