Trailblazers in RNA Biology and Splicing to Accelerate Breakthroughs in RNA Exon Editing Therapeutics
BOSTON, May 6, 2025 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, today announced the formation of its Scientific Advisory Board (SAB). These globally recognized leaders in RNA biology and gene expression will provide their collective expertise on scientific direction, translational priorities, and research strategy as Ascidian builds an industry leading portfolio of RNA editing medicines.
"We're honored to have such a distinguished group of scientific leaders join our efforts," said Michael Ehlers, MD, PhD, President and Chief Executive Officer of Ascidian Therapeutics. "Each brings a unique lens on RNA biology—from structural insights to splicing, disease mechanisms, and therapeutic translation. Their guidance will be invaluable as we pursue potentially transformative therapies for patients."
"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 Roy Parker, PhD, Distinguished Professor of Biochemistry at the University of Colorado Boulder and member of Ascidian's Scientific Advisory Board. "I'm excited to collaborate with Ascidian and fellow SAB members to help shape Ascidian's scientific direction, explore disease-specific applications, and advance therapeutics with the potential to redefine what's possible in RNA medicine."
Ascidian's RNA exon editors precisely reprogram RNA at the kilobase scale in vivo—without the use of foreign enzymes or introducing genomic risk—to address the underlying causes of disease. With the first-ever RNA exon editor in clinical development and a focused pipeline in high unmet need areas, Ascidian is expanding the potential of RNA medicine to treat diseases not addressed by today's gene editing technologies.
Members of Ascidian's Scientific Advisory Board include:
- Mariano A. Garcia-Blanco, MD, PhD, F. Palmer Weber Professor and Chair of Microbiology, Immunology, and Cancer Biology at the University of Virginia. A pioneering figure in RNA biology and RNA virology, Dr. Garcia-Blanco's research has advanced the field's understanding of RNA splicing, with foundational work in RNA trans-splicing dating back to the 1990s. Over the past three decades, he has published more than 200 papers, trained over 60 scientists, and helped shape RNA research through both academic leadership and industry innovation. He is an elected member of leading academic and scientific institutions such as the National Academy of Sciences, the American Academy of Arts and Sciences, and the American Academy of Microbiology. In addition, he is a scientific founder of multiple biotech companies and a respected voice in RNA-based therapeutic development.
- Brenton R. Graveley, PhD, Professor and Chair of Genetics and Genome Sciences at UConn Health. Dr. Graveley is an internationally recognized expert in RNA biology, known for his pioneering work on alternative splicing and RNA-binding proteins. He has played key leadership roles in major genomic efforts including ENCODE and modENCODE, and has published more than 200 papers. A fellow of the American Association for the Advancement of Science and member of the Connecticut Academy of Science and Engineering, Dr. Graveley also served on the Board of Directors of the RNA Society and was an editor of RNA.
- Roy Parker, PhD, Distinguished Professor of Biochemistry at the University of Colorado Boulder, Director of the BioFrontiers Institute, and Investigator of the Howard Hughes Medical Institute. A globally recognized leader in RNA biology, Dr. Parker's research has transformed understanding of mRNA metabolism, ribonucleoprotein particle (RNP) granules, and RNA's role in neurodegenerative disease. His discoveries—including the identification of P-bodies—have shaped the field's view of RNA regulation and decay. A member of the National Academy of Sciences and former president of the RNA Society, Dr. Parker has served as a scientific co-founder of two start-ups, and is widely regarded as one of the most influential voices in post-transcriptional gene regulation.
- Silvi Rouskin, PhD, Assistant Professor of Microbiology at Harvard Medical School. Dr. Rouskin is an RNA biologist recognized for her groundbreaking work on RNA structure and its impact on gene expression, including the discovery of alternative RNA conformations that regulate splicing. Dr. Rouskin earned her PhD at UCSF, launched her lab at MIT as a prestigious Whitehead Fellow at age 30, and now leads her research at Harvard Medical School. Dr. Rouskin is a Pew Scholar and recipient of numerous prestigious awards, including the NIH New Innovator Award and the Vilcek Prize for Creative Promise in Biomedical Science. Her work spans structural biology, virology, and therapeutic design, and she is widely regarded as one of the most innovative voices in RNA science today.
About Ascidian Therapeutics
Ascidian Therapeutics, an ATP company, 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 discovery, preclinical, and clinical 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.
Ascidian was named both an Endpoints 11 and Fierce 15 company for 2024.
For more information, visit www.ascidian.com.
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SOURCE Ascidian Therapeutics
