Ascidian and Lilly Enter Global Research Collaboration to Develop RNA Exon Editors for Devastating Kidney Diseases
Combines Ascidian's RNA exon editing product engine with Lilly's leadership in genetic medicines
Programs target undisclosed inherited kidney diseases, with Lilly option to expand to additional targets
BOSTON, June 3, 2026 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, today announced a global research collaboration and licensing agreement with Eli Lilly and Company ("Lilly") to discover and develop therapies for undisclosed monogenic kidney diseases, with the option to expand to additional targets.
Ascidian's RNA exon editors are capable of editing multiple whole exons at the kilobase scale to repair genetic instructions causing disease. Designed to address large genes or genes with high mutational variance, Ascidian's RNA exon editors expand the boundaries of genetic medicines.
"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. "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 agreement, Lilly will receive exclusive, target-specific rights to Ascidian's RNA exon editing technology for undisclosed kidney disease targets. Ascidian will lead discovery and selected preclinical activities, with Lilly responsible for additional preclinical work, clinical development, manufacturing, and commercialization. Ascidian is eligible to receive up to $1.9 billion, including an upfront payment, development and commercial milestone payments, and tiered royalties on commercial sales worldwide. Ascidian retains the right to pursue other targets in the kidney independently or with additional partners.
More than 60 genetic diseases are known to affect the kidneys, and over 3.5 million Americans live with severe inherited kidney disease today.i,ii These diseases are often caused by mutations in large genes, or genes with heterogeneous mutations, that are not addressable with existing technologies. By rewriting RNA using the cell's natural RNA splicing machinery — without modifying the genome or introducing foreign enzymes — Ascidian's RNA exon editors are designed to combine the durability of gene therapy while sharply reducing risks associated with direct DNA editing and gene replacement in these conditions.
About Ascidian Therapeutics
Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With partnered and wholly owned programs in retinal, renal, neurological, neuromuscular, and genetically defined diseases, Ascidian's approach has the potential to treat patients with one dose of an RNA exon editor, opening new therapeutic possibilities for patients and their families who are seeking breakthroughs.
Learn more at Ascidian.com, or for Ascidian's currently enrolling clinical trials in Stargardt disease, visit AscidianClinicalTrials.com.
i American Kidney Fund. Genetic testing and counseling for kidney disease. Available at: https://www.kidneyfund.org/all-about-kidneys/tests/genetic-testing-and-counseling. Accessed May 2026.
ii Franceschini N, Feldman DL, Berg JS, Besse W, Chang AR, Dahl NK, Gbadegesin R, Pollak MR, Rasouly HM, Smith RJH, Winkler CA, Gharavi AG; NKF Genetic Testing Working Group. Advancing Genetic Testing in Kidney Diseases: Report From a National Kidney Foundation Working Group. Am J Kidney Dis. 2024 Dec;84(6):751-766. doi: 10.1053/j.ajkd.2024.05.010. Epub 2024 Jul 19. PMID: 39033956; PMCID: PMC11585423.