Ascidian Therapeutics has strengthened its leadership team and scientific guidance as it advances its pioneering RNA exon editing platform, with the company announcing key appointments and clinical progress for its lead program targeting Stargardt disease.
The Boston-based biotechnology company recently formed a Scientific Advisory Board comprising four internationally recognized experts in RNA biology and splicing. The board includes Dr. Mariano Garcia-Blanco from the University of Virginia, Dr. Brenton Graveley from UConn Health, Dr. Roy Parker from the University of Colorado Boulder, and Dr. Silvi Rouskin from Harvard Medical School.
"Ascidian's RNA exon editing platform offers a novel and promising way to harness the body's natural RNA splicing machinery—enabling the precise rewriting of thousands of RNA bases with a single therapeutic molecule," said Dr. Roy Parker, Distinguished Professor of Biochemistry at the University of Colorado Boulder and Howard Hughes Medical Institute Investigator.
Leadership Expansion and Clinical Focus
In a significant leadership addition, Ascidian appointed Dr. Murray Abramson as Chief Development Officer. Dr. Abramson brings over 25 years of clinical development experience, having successfully led the filing of more than 6 NDAs and BLAs, including SPINRAZA® and TECFIDERA®. He previously held senior positions at Precision Biosciences, Tempus A.I., Biogen, and Merck.
"He brings an exceptional track record in clinical innovation, global development, and operational rigor across some of the world's leading biopharma companies," said Dr. Michael Ehlers, President and CEO of Ascidian Therapeutics. "His leadership will be instrumental as we continue unlocking the therapeutic potential of RNA exon editing across a range of devastating diseases."
The company also established an Ophthalmology Clinical Advisory Board featuring seven globally recognized retinal disease experts to guide its lead clinical program, ACDN-01, for Stargardt disease and other ABCA4 retinopathies.
Breakthrough RNA Editing Technology
Ascidian's RNA exon editors represent a novel therapeutic approach that enables precise, kilobase-scale editing of RNA directly in vivo without introducing foreign enzymes or altering the genome. This technology addresses the underlying causes of disease by reprogramming RNA at unprecedented scale, potentially treating conditions that have eluded conventional gene editing technologies.
The platform's unique mechanism offers advantages for treating diseases caused by large, mutation-rich genes where traditional gene replacement or base editing approaches face limitations due to gene size or high mutational variance.
Clinical Progress in Stargardt Disease
ACDN-01, the first RNA exon editor in clinical development, is currently being evaluated in the Phase 1/2 STELLAR trial (NCT06467344) for Stargardt disease and other ABCA4 retinopathies. The STELLAR trial represents the most advanced clinical program targeting the underlying cause of Stargardt disease.
Stargardt disease affects approximately 30,000 individuals in the United States and is the most common form of inherited macular degeneration. The condition is caused by mutations in the ABCA4 gene, leading to progressive retinal degeneration and vision loss that typically begins in childhood and young adulthood. More than 1,000 mutations across the ABCA4 gene have been identified as causing Stargardt disease, and no FDA-approved treatments currently exist.
"RNA exon editing represents an elegant approach to diseases like Stargardt that involve large, mutation-rich genes such as ABCA4," said Dr. Mark Pennesi, Director of the Inherited Retinal Degeneration Division at the Retina Foundation of the Southwest and Clinical Advisory Board member. "By reprogramming RNA directly, this modality has the potential to overcome key limitations of traditional gene therapies."
ACDN-01 has demonstrated efficient, durable in vivo RNA exon editing in non-human primate retina and ex vivo RNA exon editing in human retinal explants. The therapy is delivered as an in vivo RNA exon editor using a single vector approach.
Expanding Therapeutic Pipeline
Beyond its lead ophthalmology program, Ascidian is developing discovery, preclinical, and clinical programs across retinal, neurological, neuromuscular, and genetically defined diseases. The company's approach has the potential to treat patients with a single dose of an RNA exon editor, opening new therapeutic possibilities for patients and families seeking breakthrough treatments.
The company was recognized as both an Endpoints 11 and Fierce 15 company for 2024, highlighting its innovative approach to RNA medicine and therapeutic potential across multiple disease areas.
