Ascidian Therapeutics Appoints Dr. Murray A. Abramson as Chief Development Officer and Establishes Ophthalmology Clinical Advisory Board to Advance ACDN-01 for Treatment of Stargardt Disease
BOSTON, June 24, 2025 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, today announced the appointment of Murray A. Abramson, MD, MPH, as Chief Development Officer. The company also announced its inaugural Ophthalmology Clinical Advisory Board, established to help guide its lead clinical stage program, ACDN-01, for people living with Stargardt disease and other ABCA4 retinopathies.
With over 25 years of experience in clinical development and operational leadership, and the successful filing of more than 6 NDAs and BLAs including SPINRAZA® and TECFIDERA®, Dr. Abramson will oversee clinical strategy and execution across Ascidian's pipeline of first- and best-in-class RNA editing therapies. The newly established Ophthalmology Clinical Advisory Board is comprised of seven globally recognized leaders in retinal disease who will provide expert guidance on clinical strategy, trial design, and therapeutic development in support of Ascidian's lead ACDN-01 program.
"We are thrilled to welcome Murray to Ascidian as Chief Development Officer," said Michael Ehlers, MD, PhD, President and Chief Executive Officer of Ascidian Therapeutics. "He brings an exceptional track record in clinical innovation, global development, and operational rigor across some of the world's leading biopharma companies. His leadership will be instrumental as we continue unlocking the therapeutic potential of RNA exon editing across a range of devastating diseases."
Dr. Ehlers continued, "This outstanding group of advisors brings unparalleled expertise in retinal disease and will play a critical role in shaping the clinical development path for our lead program. Their insights and expertise will help us advance our efforts to transform outcomes for people living with Stargardt disease and other retinal disorders."
Ascidian's RNA exon editors enable precise, kilobase-scale editing of RNA directly in vivo without introducing foreign enzymes or altering the genome. This novel mechanism offers a new way to target the root causes of disease, including those that have eluded conventional gene therapies.
The company's lead candidate, ACDN-01, is the first RNA exon editor in clinical development and is currently being evaluated in the Phase 1/2 STELLAR trial (NCT06467344) for the treatment of Stargardt disease and other ABCA4 retinopathies. STELLAR is the most advanced clinical program targeting the underlying cause of Stargardt disease.
"With its groundbreaking RNA exon editing product engine, Ascidian is expanding the boundaries of RNA medicine," said Dr. Abramson. "I'm honored to join the team and excited to help drive ACDN-01 and Ascidian's full pipeline forward. Working alongside a world-class team of scientists and advisors, I look forward to translating groundbreaking science into meaningful clinical outcomes."
Mark Pennesi, MD, PhD, Director of the Inherited Retinal Degeneration Division at the Retina Foundation of the Southwest and a member of the Clinical Advisory Board added, "RNA exon editing represents an elegant approach to diseases like Stargardt that involve large, mutation-rich genes such as ABCA4. By reprogramming RNA directly, this modality has the potential to overcome key limitations of traditional gene therapies. Together with my colleagues, I look forward to collaborating with Ascidian to help shape the clinical path for this exciting therapeutic innovation."
About Murray A. Abramson, MD, MPH
Dr. Abramson is a medical internist and infectious disease specialist with deep expertise in drug development and clinical operations. Over the course of his career to date, Dr. Abramson has led the successful filing of more than 6 NDAs and BLAs including SPINRAZA® and TECFIDERA®. He most recently served as SVP, Head of Clinical Development at Precision Biosciences and previously held senior leadership roles at Tempus A.I., Biogen, and Merck. At Biogen, he led Global Clinical Operations for nearly a decade, and at Merck, he spent over 12 years in medical research and clinical operations. Before entering the biopharma industry, Dr. Abramson was a faculty member at Duke University School of Medicine and led infectious diseases research at the Duke Clinical Research Institute. He earned his MD from Duke University Medical Center and MPH in Epidemiology from the University of North Carolina at Chapel Hill.
Members of Ascidian's Ophthalmology Clinical Advisory Board include:
Karl Csaky, MD, PhD, is Chief Executive and Medical Officer at the Retina Foundation of the Southwest and the T. Boone Pickens Director of the Molecular Ophthalmology Laboratory and Clinical Center of Innovation for AMD where he leads research to advance the understanding and treatment of age-related macular degeneration (AMD) and other forms of macular degeneration. With more than 25 years of experience in drug development and translational research, Dr. Csaky is recognized for his work on AMD pathogenesis and therapeutic innovation. A former Investigator at the National Eye Institute, he directed the Laboratory of Retinal Diseases and Therapeutics and has led numerous clinical trials in AMD and other retinal degenerations. A previous Fulbright Scholar, Dr. Csaky completed both a retina fellowship at the Wilmer Eye Institute at Johns Hopkins and a post-doctoral fellowship at the National Cancer Institute.
Christine Kay, MD is Director of Electrophysiology and Retinal Genetics, and Director of Clinical Research at Vitreoretinal Associates in Gainesville, Florida. She graduated magna cum laude from Harvard University with a degree in neuroscience and went to medical school at the University of Florida. She completed her ophthalmology residency at the University of South Florida where she was chief resident, and completed her vitreoretinal fellowship training at the University of Iowa. During her fellowship at University of Iowa, she developed a particular interest in inherited retinal disease (IRD). A vitreoretinal surgeon and IRD specialist, Dr. Kay currently serves as principal investigator in 30 clinical trials, with a particular focus and interest in inherited retinal dystrophy trials, gene therapy, and optimization of surgical subretinal delivery techniques. Dr. Kay launched her research career with a five-year Career Development Award from the Foundation Fighting Blindness and previously was chief of the retinal genetics service and vitreoretinal fellowship at the University of Florida. She has authored more than 20 peer-reviewed publications, contributes to leading ophthalmology journals, and is a member of the Retina Society and Macula Society. She is an active Foundation Fighting Blindness Consortium principal investigator, and has active advisory roles in several IRD biotech companies, engaging with FDA and regulatory aspects of clinical development of novel therapies.
Byron L. Lam, MD, is Professor and Mark J. Daily Chair of Ophthalmology at the University of Miami Miller School of Medicine's Bascom Palmer Eye Institute. A leading authority in IRDs and neuro-ophthalmology, Dr. Lam directs the Daily Research Center for IRD and is the medical director of Clinical Research at Bascom Palmer. He has pioneered numerous clinical trials, including the first U.S. gene therapy study for choroideremia, and has led research into treatments for retinitis pigmentosa, achromatopsia, and Stargardt disease. He developed the Visual Light Sensitivity Questionnaire-8 (VLSQ-8) and co-created the Bascom Palmer Light Discomfort Analyzer to improve clinical trial endpoints. Dr. Lam serves on the editorial board of the American Journal of Ophthalmology and is a recipient of the Senior Achievement Award from the American Academy of Ophthalmology.
Bart Leroy, MD, PhD, is Professor of Ophthalmology, Ophthalmic Genetics, and Visual Electrophysiology at Ghent University and Head of the Department of Ophthalmology at Ghent University Hospital. A board-certified ophthalmologist and clinical geneticist, Professor Leroy is internationally recognized for his expertise in IRDs, with a particular focus on deep phenotyping and the development of gene-based therapies. He played a key clinical role in the development and approval of Luxturna®, the first FDA- and EMA-approved gene therapy for an inherited disease. His academic and clinical training includes fellowships at Moorfields Eye Hospital and University College London, with additional appointments at the Children's Hospital of Philadelphia and the University of Pennsylvania. Professor Leroy is a Senior Clinical Investigator of the Research Foundation Flanders and is a member and Board member of several professional organizations including ISGEDR, ERN-EYE, SGOF and EURETINA, as well as a past president of the European Organization for Vision & Eye Research.
Michel Michaelides, MB BS, MD(Res), FACS is Professor of Ophthalmology at the UCL Institute of Ophthalmology and a Consultant Ophthalmologist at Moorfields Eye Hospital, where he specializes in medical retina, inherited eye disease, and pediatric ophthalmology. A global leader in IRDs, Professor Michaelides has helped define disease mechanisms and endpoints in conditions such as Stargardt disease, retinitis pigmentosa, and USH2A-retinopathy, while advancing translational research through novel imaging techniques and targeted therapeutic development. Professor Michaelides has authored more than 500 peer-reviewed publications and 55 book chapters, and serves as principal investigator for more than 10 interventional studies. He is a member of the Macula Society and Retina Society, and a member of the Foundation Fighting Blindness Clinical Consortium Executive Committee.
Mark Pennesi, MD, PhD, FARVO, is Director of the Inherited Retinal Degeneration Division at the Retina Foundation of the Southwest and an adjunct Professor of Ophthalmology at the Paul H. Casey Ophthalmic Genetics Division at the Casey Eye Institute. A clinician-scientist recognized for his leadership in IRDs, Dr. Pennesi has played a central role in advancing the field's clinical research infrastructure and therapeutic pipeline. He serves as principal investigator or co-investigator on a broad portfolio of clinical trials, including gene augmentation, gene editing, and therapies for conditions such as achromatopsia, X-linked retinitis pigmentosa, and CEP290- and ABCA4-related retinopathies. Dr. Pennesi is a member of the Macula Society and Retina Society, and the recipient of career development awards from the Foundation Fighting Blindness and Research to Prevent Blindness.
Katarina Stingl, MD, is Professor of Ophthalmology at the University of Tübingen, where she leads both the Center for Rare Eye Diseases and the Clinical Unit for Inherited Retinal Degenerations. An internationally recognized expert in clinical and genetic diagnostics for IRDs, Dr. Stingl also heads a research group focused on multimodal retinal diagnostics, including electrophysiology, functional and metabolic retinal imaging, and advanced retinal imaging techniques. She serves as Principal Investigator or Co-Principal Investigator on more than 15 clinical trials in IRDs and brings deep expertise in genetic therapies, visual implants, pharmacotherapy, and electrostimulation. Dr. Stingl is an active member of ERN-EYE.
About Stargardt Disease and ACDN-01
Stargardt disease is the most common form of inherited macular degeneration and has no FDA approved treatments. Affecting approximately 30,000 individuals in the U.S. alone, Stargardt disease is caused by mutations in the ABCA4 gene which lead to progressive retinal degeneration and vision loss, typically beginning in childhood and young adulthood.
More than 1,000 mutations across the ABCA4 gene have been found to cause Stargardt disease. Diseases caused by ABCA4 loss of function – including Stargardt disease – are examples of genetic disorders that cannot be addressed by standard gene replacement, given the large size of the gene, or by base editing, due to the high mutational variance of the affected gene.
ACDN-01 is an in vivo RNA exon editor delivered by a single vector. It has demonstrated efficient, durable in vivo RNA exon editing in non-human primate retina and ex vivo RNA exon editing in human retinal explants.
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.