In this episode of "Rare Insights," host Taren Grom speaks with Dr. Jay Barth, Chief Medical Officer at Ascidian Therapeutics, about groundbreaking advancements in RNA editing technology and its potential in the rare disease space. Dr. Barth discusses Ascidian’s innovative approach, inspired by “sea squirts,” and shares insights on the development of ACDN-01 for Stargardt disease, an inherited form of macular degeneration. This episode also explores the challenges and opportunities in rare disease drug development and the unique aspects that draw professionals to this field.

Discussion Topics:

1. Introduction to Ascidian’s Technology: Exploring the innovative RNA editing inspired by "sea squirts" and its implications for treating rare diseases.
2. Reducing Risks in Gene Editing: How Ascidian’s RNA exon editing approach minimizes risks compared to traditional gene editing methods.
3. FDA Fast-Track Designation for ACDN-01: Insights on achieving fast-track status for Stargardt disease treatment and future plans for Ascidian’s pipeline.
4. RNA Editing Momentum: The pressures and focus brought by the increasing interest in RNA editing.
5. Personal Journey in Rare Diseases: Dr. Barth shares what inspired his career in the rare disease sector and what continues to excite him about this work.

Dr. Jay Barth is Chief Medical Officer of Ascidian Therapeutics. 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. 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.

Stay tuned for the next new episode of Rare Insights! In the meantime, listen to all our episodes Apple Podcasts, Spotify, streaming on the website, or any other podcast player by searching, “Rare Insights”.

Rare Insights’ team includes Taren Grom, Liz Kay, Kaitlyn Taylor, DNA Today’s Kira Dineen, and Nina Wachsman.

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