BOSTON, April 28, 2026 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, today announced it will present two oral presentations and one poster presentation at the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Boston, MA, May 11-15, 2026.
Across two oral presentations, Ascidian will feature the first-ever human safety data for its lead RNA exon editing candidate, ACDN-01, from the Phase 1/2 STELLAR trial in Stargardt disease, the most common form of inherited blindness. Additionally, the company will present new data demonstrating how its AI/ML-enabled RNA discovery platform drives efficient identification and optimization of RNA exon editors across diverse genetic targets.
In a poster presentation, Ascidian scientists will also share preclinical data supporting MSH3 splice modulation as a potential therapeutic strategy for repeat expansion disorders, a broad group of genetic disorders including Huntington's disease, spinocerebellar ataxia, myotonic dystrophy, and others. It highlights a novel targeted RNA approach designed to modulate disease-driving biology at its source.
Together, these presentations underscore both the clinical momentum of Ascidian's lead program and the expanding reach of its platform across multiple therapeutic areas.
"The data we are presenting at ASGCT reflect both meaningful clinical progress and the growing versatility of our platform," said Michael Ehlers, M.D., Ph.D., Founder, President and Chief Executive Officer of Ascidian Therapeutics. "From our first clinical insights in Stargardt disease to emerging approaches targeting repeat expansion biology, we are reaching beyond conventional genetic medicines to provide new therapeutic possibilities."
Presentation details
Session: Preclinical translational large animal studies
Presentation ID: 115
Oral Presentation: RNA Exon Editing for Stargardt Disease: Non-Clinical Development and First-in-Human Clinical Safety of ACDN-01
Date: Tuesday, May 12
Time: 3:45 - 4:00 PM
Location: Room 258ABC (Level 2), Thomas M. Menino Convention & Exhibit Center
Session: Expanding cell and gene therapy through therapeutic RNA and epigenome engineering
Presentation ID: 499
Oral Presentation: Discovery of RNA Exon-Editing Therapeutics Across Diverse Genetic Diseases via High-Throughput Screening, Enabling Novel Machine Learning Integration
Date: Friday, May 15
Time: 4:15 - 4:30 PM
Location: Room 052AB (Exhibit Level), Thomas M. Menino Convention & Exhibit Center
Session: Poster Reception
Presentation ID: 2471
Poster Presentation: MSH3 Pre-mRNA Splice Modulation in the Treatment of Repeat Expansion Disorders
Date: Wednesday, May 13
Time: 5:00 - 6:30 PM
Location: Exhibit and Poster Hall (Halls B2-C, Exhibit Level), Thomas M. Menino Convention & Exhibit Center
In addition, Ascidian SVP of Operations, John Kerwin, Ph.D., will participate in a symposium hosted by Forge Biologics, "More Doses, Same Run: Process Innovation and Late-Phase Manufacturing Frameworks for AAV Developers," taking place on Wednesday, May 13 at 10:15 AM. Ascidian will showcase the process innovations that drove significant ACDN-01 batch productivity gains, establishing a robust, scalable manufacturing process to support late-phase clinical supply.
Presentation materials will be available upon request via the Publications & Presentations section of the Ascidian website following the live presentations.
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, 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 www.ascidian.com, or for more information about STELLAR and STARPATH, Ascidian's clinical trials in Stargardt disease, visit www.AscidianClinicalTrials.com.
View original content to download multimedia:https://www.prnewswire.com/news-releases/ascidian-to-highlight-progress-of-lead-program-in-stargardt-and-platform-advancements-across-therapeutic-areas-at-asgct-2026-annual-meeting-302755028.html
SOURCE Ascidian Therapeutics
