Eli Lilly signed a collaboration and licensing agreement with Boston-based Ascidian Therapeutics on Wednesday to research and develop treatments for inherited kidney diseases, with Ascidian eligible to receive up to $1.9 billion under the deal.
As part of the agreement, Ascidian granted Lilly rights to its RNA exon-editing platform that are both exclusive and tied to specific targets in undisclosed kidney diseases, the company said
Responsibilities are divided between the two companies, with Ascidian taking the lead on early-stage discovery and some preclinical activities, and Lilly stepping in for later preclinical work, clinical trials, manufacturing, and bringing any resulting products to market. The deal also gives Lilly the flexibility to bring additional disease targets into the partnership, and Ascidian is free to advance other kidney disease programs on its own or through separate arrangements. The total potential value of $1.9 billion is made up of an initial payment at signing, future payments contingent on development and commercial benchmarks, and royalties that scale with global sales, the company said.
Ascidian’s technology edits multiple whole exons at the RNA level to repair genetic instructions that cause disease, without permanently modifying a patient’s DNA. More than 60 genetic diseases are known to affect the kidneys, and over 3.5 million Americans live with severe inherited kidney disease, the company said. “RNA exon editing gives us the ability to rewrite genes at their source, without altering DNA, opening the door to diseases long out of reach,” Ascidian CEO Michael Ehlers said in a statement. Speaking to Reuters, Ascidian chief business officer Daniel Rosan described the platform as one that captures the core advantages of CRISPR and other DNA-based approaches, but sidesteps their most significant drawback by leaving the patient’s underlying genetic code intact.