Ascidian Partners with Eli Lilly on RNA Editing for Monogenic Kidney Targets
A newly signed USD 1.9 billion global research collaboration and licensing agreement is set to combine Ascidian Therapeutics’ exon editing platform with Eli Lilly’s expertise in genetic medicines.
RNA-editing specialist biotechnology company, Ascidian Therapeutics, has announced that it has entered into a global research collaboration and licensing agreement with Eli Lilly and Company. The agreement, which was announced in a June 3, 2026 press release (1), will see the companies develop RNA exon editors for monogenic kidney diseases.
The RNA exon editing platform, developed by Ascidian, replaces mutated exons with wild-type ones through high-throughput molecular biology in tandem with computational biology. As the exon editing takes place at the RNA level, potential risks of off-target DNA edits of the expression of transgenes in inappropriate cell types is minimized (2).
“Lilly and Ascidian believe that patients with serious monogenic kidney diseases deserve effective treatment options and that an RNA-based approach is a compelling strategy for those diseases,” said Michael Ehlers, M.D., Ph.D., President and Chief Executive Officer of Ascidian Therapeutics, in a company press release (1). “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. Combined with Lilly’s genetic medicine expertise, we aim to dramatically reduce the burden of genetic kidney disease.”
Under the terms of the agreement, Lilly will gain exclusive, target-specific rights to Ascidian’s RNA exon editing platform for undisclosed kidney disease targets. The discovery activities and some of the selected preclinical activities will be under the remit of Ascidian, but Lilly will be responsible for additional preclinical work, clinical development, manufacturing, and commercialization.
Through the deal, Ascidian is eligible to receive up to USD 1.9 billion, comprising an upfront payment, development and commercial milestone payments, and tiered royalties on commercial global sales. However, Ascidian retains the rights to pursue other targets in the kidney independently or with other partners.
At the beginning of the year, Ascidian formed a manufacturing partnership with Forge Biologics, aimed at advancing RNA editing therapy for Stargardt disease, ACDN-01. Through the partnership, Forge is providing Ascidian with process and analytical development services, toxicology, and current GMP manufacturing for ACDN-01, while Ascidian is using Forge’s proprietary FUEL technologies and program-specific optimizations to drive manufacturing efficiencies.
“We are proud to partner with Ascidian Therapeutics on this program, which reflects our shared commitment to bringing together scientific and manufacturing innovation to support patients,” commented John Maslowski, President and Chief Executive Officer of Forge, in a press release about the partnership (3). “Forge’s FUEL platform technologies and manufacturing capabilities were built to enable partners like Ascidian as they advance their work through clinical development and beyond.”
“ACDN-01 represents a fundamentally new approach to treating diseases driven by large, complex genes like ABCA4 which have historically been difficult to address with traditional gene therapies,” added Michael Ehlers, MD, PhD, President and Chief Executive Officer of Ascidian, in the press release (3). “Partnering with Forge Biologics equips us with deep AAV expertise, scalable manufacturing capabilities, and significant capacity, enabling us to rapidly advance ACDN-01 into late-stage clinical development.”
References
Ascidian. Ascidian and Lilly Enter Global Research Collaboration to Develop RNA Exon Editors for Devastating Kidney Diseases. Press Release, June 3, 2026.
Ascidian. Rewriting RNA. Ascidian-tx.com, accessed June 3, 2026.
Ascidian. Ascidian Therapeutics and Forge Biologics Announce Manufacturing Partnership to Advance RNA Editing Therapy for Stargardt Disease. Press Release, Jan. 7, 2026.