In Episode 3 of our in vivo-focused special series of Cell & Gene: The Podcast, Ascidian Therapeutics' Founder, President, and CEO Mike Ehlers, M.D., Ph.D., explains how the company is advancing a new class of genetic medicines built on RNA exon editing to durably correct disease at the transcript level without permanently altering DNA. He also shares why in vivo therapies still need to prove precision, durability, and predictability before they become mainstream, and why the field must move beyond proof of concept to reproducible clinical performance across patients and diseases. He discusses the importance of patient selection, tissue specificity, and matching editing efficiency to biological thresholds, and much more.
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