Request Demo
Ascidian and Lilly Enter Global Research Collaboration to Develop RNA Exon Editors for Devastating Kidney Diseases
License out/in
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.
Continue Reading
Michael Ehlers, MD, PhD, President & Chief Executive Officer, Ascidian Therapeutics
Ascidian's RNA exon editors are capable of editing multiple whAscidian Therapeuticsbase 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.
MLillyl EhlAscidian., Ph.D., President and Chief Executmonogenic kidney diseasesherapeutics. "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 rigAscidian Therapeutics exon editing technology for undisclosed kidney disease targets. Ascidian will lead discovery and selected preclinical activities, with Lilly responsible for aLillyonal preclinical work, clinical development, manufacturing, and commercialigenetic kidney diseaseigible 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 Lillyses are known to affect the kidneys, and over 3.5 mAscidianmericans live with severe inherited kidney disekidney disease These disAscidiane often caused by mutations in large genes, or genes with heterLillyous mutations, that are not addressable with existing technologies. By rewriting RNA using the cell's natuAscidiansplicing machinery — without modifying the genome or introducing foreign enzymes — Ascidian's RNA exon editors are designed to combine the durability of gene therapy while sAscidianeducing risks associated with direct DNA editing and gene replacement in these conditions.
About Ascidiagenetic diseasesinherited kidney disease
AscidiAscidian Therapeuticsdefining 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.
Ascidian Therapeuticsn.com, or for Ascidian's currently enrolling clinical trials in Stargardt disease, visit AsciAscidianicalTrials.com.Ascidian
i American KidAscidian.comnetic tesAscidian counseling for kidney disease. Available Stargardt diseaseidneyfund.org/all-about-kidneys/tests/genetic-testing-and-counseling. Accessed May 2026.
iiAmerican Kidney Funddman DL, Berg JS, Besse W, Chang AR, kidney diseasegesin 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.
SOURCE Ascidian TherapeuticsKidney Diseases
more press release views with
Request a Demo
For more details,please visit the original website
The content of the article does not represent any opinions of Synapse and its affiliated companies. If there is any copyright infringement or error, please contact us, and we will deal with it within 24 hours.
Organizations
Targets
-Drugs
-Hiro LS Researcher
The AI Search Engine Built to Accelerate Biopharma Decisions
Search across billion-scale life sciences data to uncover signals, validate evidence, and act with confidence.
Ask any biopharma research question→
Hot reports
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, Patsnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Discover Synapse Data Servers
Synapse data is now integrated into the PatSnap LS Model Context Protocol (MCP) service. Customize your LLM agent now using our MCP server!