Ascidian Therapeutics Appoints Jay A. Barth, M.D., as Chief Medical Officer

Boston — Ascidian Therapeutics, a biotechnology company focused on treating human diseases by rewriting RNA, announced the appointment of Jay A. Barth, M.D., as Chief Medical Officer (CMO). With 25 years of experience in drug development and medical affairs, and a focus in gene therapy and rare diseases, Dr. Barth will be responsible for all clinical development activities across Ascidian’s diversified pipeline in ophthalmology, neurological, neuromuscular, and genetically defined diseases.

“Jay’s deep knowledge and experience in gene therapy and complex indications will make an immense impact for patients and for Ascidian, as we advance our exon editing therapies addressing the underlying causes of disease,” said Romesh Subramanian, Ph.D., President and Chief Executive Officer of Ascidian Therapeutics. “Jay brings a breadth of expertise across clinical development, medical, and regulatory affairs that is perfectly suited to advance our lead programs into the clinic and help us achieve our vision of delivering transformative therapies to patients by rewriting RNA. We’re thrilled to welcome Jay to Ascidian.”

Dr. Barth has wide-ranging clinical development expertise across many challenging therapeutic categories. He led the clinical efforts of the Marketing Authorization Application (MAA) process leading to the first regulatory approval of a treatment for Duchenne muscular dystrophy while at PTC Therapeutics. As CMO at Amicus Therapeutics, he oversaw the clinical components of the EU marketing application which culminated in the first oral treatment of Fabry disease. Dr. Barth has also worked extensively with regulatory affairs teams in the U.S. and EU to develop regulatory strategies, liaise with regulatory agencies, and prepare marketing applications.

Early in his career, Dr. Barth developed drugs for broad patient populations, primarily in the areas of gastrointestinal and pulmonary diseases, at companies including Eisai Medical Research and Merck. His subsequent drug development work shifted to rare diseases and then specifically to gene therapy. Immediately prior to joining Ascidian, Dr. Barth served as CMO for Lexeo Therapeutics, a clinical-stage gene therapy company focused on addressing cardiovascular and central nervous system diseases. At Lexeo, he led the team’s efforts in the first IND cleared by the FDA for a gene therapy targeting Friedreich’s ataxia cardiomyopathy.

“The opportunity to be part of a company with the potential to redefine the treatment of genetic diseases is what drew me to Ascidian Therapeutics,” said Dr. Barth. “Ascidian’s unique exon editing platform offers a promise to address conditions beyond the reach of current gene therapies and gene editing approaches, and consequently brings new hope to people who have limited or no treatment options today. I’m excited to contribute my expertise to help make that promise a reality for patients.”

Dr. Barth received his M.D. from the University of Pennsylvania School of Medicine and his B.A. from Columbia University. He has authored numerous publications in peer-reviewed journals and is an active member of the American Society of Gene & Cell Therapy, American Society of Human Genetics, and the European Society of Gene & Cell Therapy.

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 active discovery and preclinical programs in ophthalmology, neurological, neuromuscular, and genetically defined diseases, Ascidian’s approach has the potential to treat patients with a single dose of a single exon-editing RNA therapeutic and is opening new therapeutic possibilities for patients in need of breakthroughs. For more information, visit