#United States #Boston #Stargardt Disease #Ascidian Therapeutics #ACDN-01

Ascidian Therapeutics Showcases Breakthroughs at ASGCT 2026

Ascidian Therapeutics, a pioneering biotechnology firm dedicated to combating human diseases through RNA editing, is gearing up to present exciting developments at the upcoming American Society of Gene & Cell Therapy (ASGCT) annual meeting in Boston, MA, from May 11-15, 2026. The company will conduct two oral presentations and one poster presentation highlighting their innovative programs focused on Stargardt disease and other genetic disorders.

First Presentation: ACDN-01 in Stargardt Disease

In one of the notable oral presentations, Ascidian will introduce the first safety data from its lead RNA exon editing therapy, ACDN-01. This important information comes from the Phase 1/2 STELLAR trial, which focuses on Stargardt disease, recognized as the most prevalent inherited cause of blindness. The data marks a significant milestone in Ascidian's commitment to delivering transformative treatments for patients suffering from genetic conditions.

Second Presentation: AI-Driven RNA Discovery

Alongside the patient data, the company will present groundbreaking findings showcasing how its artificial intelligence and machine learning-enabled RNA discovery platform enhances the identification and optimization of RNA exon editors. This new approach broadens the potential for targeting diverse genetic disorders, ensuring that advancements in gene therapies keep pace with emerging medical challenges.

Poster Presentation: Addressing Repeat Expansion Disorders

In addition to the oral presentations, Ascidian scientists will share vital preclinical results on the modulation of MSH3 pre-mRNA splicing. This strategic focus could unveil therapeutic options for several repeat expansion disorders, such as Huntington’s disease, spinocerebellar ataxia, and myotonic dystrophy. This approach emphasizes a novel RNA-targeted strategy that aims to address the underlying biological mechanisms causing these complex genetic disorders.

Insights from Leadership

Michael Ehlers, M.D., Ph.D., the Founder, President, and CEO of Ascidian Therapeutics, expressed enthusiasm about the data set for presentation. He remarked, "The data we are presenting at ASGCT reflect both meaningful clinical progress and the growing versatility of our platform. 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

• - First Oral Presentation: RNA Exon Editing for Stargardt Disease

- Date: Tuesday, May 12
- Time: 3:45 PM - 4:00 PM
- Location: Room 258ABC (Level 2) at the Thomas M. Menino Convention & Exhibit Center

• - Second Oral Presentation: Discovery of RNA Exon-Editing Therapeutics

- Date: Friday, May 15
- Time: 4:15 PM - 4:30 PM
- Location: Room 052AB (Exhibit Level) at the Thomas M. Menino Convention & Exhibit Center

• - Poster Presentation: MSH3 Pre-mRNA Splice Modulation

- Date: Wednesday, May 13
- Time: 5:00 PM - 6:30 PM
- Location: Exhibit and Poster Hall (Halls B2-C, Exhibit Level) at the Thomas M. Menino Convention & Exhibit Center

Additionally, John Kerwin, Ph.D., Ascidian's Senior Vice President of Operations, will engage in a discussion at a symposium hosted by Forge Biologics. This presentation will focus on innovative manufacturing processes that enhance productivity for AAV developers, establishing a strong foundation for late-phase clinical supply of ACDN-01.

Conclusion

Ascidian Therapeutics continues to push the envelope on how diseases can be treated by focusing on RNA editing. With their presentations set to provide a comprehensive overview of their advancements in Stargardt disease and beyond, they exemplify a commitment to meaningful progress in the field of genetic medicine. To keep updated on these developments, follow the presentations and discussions from the ASGCT conference, alongside insights from Ascidian's ongoing efforts to innovate how diseases are treated on a molecular level.