Advancing RNA Therapy for Kidneys
Eli Lilly and Ascidian partner for $1.9B RNA exon editing treatments targeting inherited kidney diseases.
Eli Lilly is partnering with Ascidian Therapeutics on a global research collaboration to develop RNA exon editing treatments for inherited kidney diseases, with the effort focused on undisclosed monogenic targets and an option to expand to additional ones. The deal is valued at up to $1.9 billion, including an undisclosed upfront payment, milestone-based payouts across research, development, and commercialization, and tiered royalties. Ascidian’s platform edits RNA by replacing disease-causing exons so the body can produce functional proteins, a strategy intended to avoid the permanent DNA changes and other risks associated with traditional gene editing. Because only the diseased exons must be replaced, the company says the payload can be sized for use in adeno-associated virus and other delivery vehicles, potentially improving reach to target tissues compared with some viral gene-therapy approaches. The companies argue the approach could move beyond current kidney disease management approaches such as symptom control, dialysis, or transplants by addressing the underlying genetic cause of disease. The partnership also reflects Lilly’s broader push deeper into genetic medicines while Ascidian emphasizes pursuing indications where it expects a differentiated clinical profile.
Key Points
Ascidian CEO Michael Ehlers said a single RNA exon editor can tackle “multiple mutations spanning multiple exons,” replacing “thousands of bases at a time” and using the cell’s own machinery—describing the work as “rewriting whole chapters at the kilobase scale,” rather than “edit[ing] letters in the genetic code.”
Ehlers also detailed the planned in vivo delivery mechanism: “The therapeutic is an AAV that expresses the designed RNA exon editor. The AAV infects the target cells. The episome of the AAV dwells in the nucleus and expresses this engineered RNA molecule… that then conducts the trans-splicing.”
Ascidian emphasizes selectivity in what it chooses to pursue. Chief financial and business officer Dan Rosan said, “We can probably edit almost any gene, but that doesn’t mean we should,” and that the company looks for areas where it can create “a really differentiated clinical profile.”
The companies framed the kidney disease need with epidemiology: they noted “more than 60 genetic diseases affect the kidneys,” and that “more than 3.5 million people in the U.S. have severe inherited kidney disease.”
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