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Leadership appointments come as Ascidian's lead program targeting ABCA4 retinopathies, including Stargardt disease, moves through IND-enabling studies BOSTON, April 11, 2023 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company focused on treating human diseases by rewriting RNA, announced today three new appointments to its growing development team as the company advances its lead program into the clinic. Alia Rashid, MBChB, joins as Vice President, Clinical Development; Sarah DiSalvatore, MPH, has been appointed Vice President, Clinical Operations; and Carmen Jacome, MBA, will serve as Vice President, Program Management. They join as Ascidian prepares for its first-in-human study in its lead program targeting ABCA4 retinopathies and progresses its diversified pipeline of programs in retinal, neurological, neuromuscular, and genetically defined diseases. "This is an exciting and very important time for Ascidian as we advance our lead program using our proprietary platform for rewriting RNA into the clinic," said Jay A. Barth, M.D., Chief Medical Officer of Ascidian Therapeutics. "We're thrilled to welcome Alia, Sarah, and Carmen – who bring deep expertise in their respective areas of focus, with experience-based knowledge from preclinical studies and all phases of clinical development through marketing authorization. I look forward to the many contributions they'll bring to Ascidian for the benefit of patients as we advance this program and move our full pipeline forward to provide breakthrough therapies that address underlying causes of disease." Ascidian's lead program targets ABCA4 retinopathies, including Stargardt disease. Diseases caused by ABCA4 loss of function represent an area of significant unmet need. More patients lose their vision from ABCA4 retinopathy than any other genetic cause, and these diseases 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. Stargardt disease is the most common form of inherited macular degeneration, affecting more than 30,000 individuals in the United States alone, and there are currently no approved treatments. By rewriting RNA, Ascidian's approach has the potential to treat patients with a single dose of a single exon-editing RNA therapeutic. Alia Rashid, MBChB, Vice President, Clinical Development Dr. Rashid joins Ascidian to help lead the clinical elements of the IND submission and oversee clinical studies in ABCA4 retinopathy. She will also help set strategic direction and manage clinical development activities for the company's overall retinal pipeline. As an ophthalmologist with extensive experience in inherited retinal diseases, Dr. Rashid brings expertise in ophthalmic pathology, ophthalmic imaging, and drug development spanning multiple modalities and indications, including AAV-mediated approaches for various retinal conditions. She was the clinical lead for the IND application and designed and oversaw clinical trials for the first functional, recombinant Complement Factor H (CFH) in dry age-related macular degeneration (dry AMD) while at Gemini Therapeutics. As Senior Director and Clinical Development Lead for Ophthalmology at Editas Medicine, Dr. Rashid helped lead clinical development for its CRISPR/Cas9 gene editing technology for patients with Leber congenital amaurosis type 10 (LCA10) – a rare genetic disease that causes blindness. She also focused on phase II and phase IV clinical studies for patients with wet AMD and diabetic retinopathy at Genentech and led pilot studies in ophthalmology innovation and mHealth to improve clinical trials. Dr. Rashid has been named to the "Top 10 Healthcare Trendsetters" and "Boston 40 Under 40 Healthcare Innovators" lists by Medtech. She completed fellowships in ophthalmic pathology at the Massachusetts Eye and Ear/Harvard Medical School and ophthalmic oncology and pathology at Emory University, and earned her medical degree from the University of Birmingham in the UK. Sarah DiSalvatore, MPH, Vice President, Clinical Operations As Vice President of Clinical Operations, Sarah DiSalvatore will be responsible for execution of Ascidian's first ever clinical trials. She brings more than 25 years of experience in end-to-end research and development and wide-ranging expertise across many challenging therapeutic categories and modalities, including both AAV and lentiviral vector (LVV) gene therapies. DiSalvatore's work has directly contributed to FDA marketing authorization of novel treatments and vaccines, including ELZONRIS™, Portrazza®, Cyramza®, Lartruvo®, Prevnar 13®, and Prevnar 7®. Most recently, DiSalvatore served as Vice President of Clinical Operations at AGTC (Applied Genetic Technologies Corporation), managing overall clinical operations for the company's gene therapy trials in rare diseases and ophthalmology. Prior to that, she was Assistant Vice President of Clinical Operations at Rocket Pharma, where she led global gene therapy clinical trials in rare diseases. DiSalvatore also has experience leading teams at both large pharmaceutical companies, such as Roche, as well as biotech startups, including Stemline Therapeutics, where she initiated six clinical trials and contributed to filing three Investigational New Drugs (IND) within three years. DiSalvatore currently serves on the Leadership Advisory Board for the DDX3X Foundation and on the Community Advocacy Committee for Simons Searchlight, an international research program whose mission is to improve the lives of people with rare genetic neurodevelopmental disorders. She earned her B.S. in biology from Salisbury University and her master's degree in public health in epidemiology from New York Medical College. Carmen Jacome, MBA, Vice President, Program Management Carmen Jacome joins Ascidian to lead the company's Program Management function, which will work cross-functionally to meet key deliverables, milestones, and timelines – from drug candidate selection, through clinical proof of concept and full development across Ascidian's diversified pipeline. She brings over 20 years of experience in project leadership including ten product launches across diverse platforms including: ophthalmology, bacterial infections, osteoporosis, multiple sclerosis, and consumer products. Most recently, while at Flexion Therapeutics, Jacome managed launch readiness and commercialization of Zilretta®, the company's first approved product. Jacome's experience encompasses both working with large pharmaceutical companies, such as Pfizer and Novartis, as well as small biotechnology startups, including Thrombogenics, Cempra, Flexion, and Astria Therapeutics. She earned her graduate certificate in program management from Stanford University and her MBA in international business from the Darla Moore School of Business at the University of South Carolina. 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 .