How Biotech Startups Use RNA Editing Without Cutting DNA

Episode 49 of Biotech Business with Fexingo explores the emerging field of RNA editing — a therapeutic approach that corrects disease-causing mutations at the RNA level without altering the genome. Hosts Lucas and Luna focus on a specific startup, Ascidian Therapeutics, and their lead candidate for Stargardt disease, a form of juvenile macular degeneration. The episode explains how RNA exon editing works, contrasting it with CRISPR-based gene editing, and discusses the regulatory landscape as the field approaches its first clinical data. Ascidian recently raised a $100 million Series A in 2024, reflecting investor enthusiasm for this safer alternative to permanent gene edits. The hosts also touch on competition from other modalities like base editing and the potential for RNA editing to treat large genes that are difficult to deliver via traditional gene therapy. A concrete example: the ABCA4 gene, which causes Stargardt disease, is too large for standard AAV vectors, but RNA editing can target it without needing to deliver the full gene. The episode concludes with the question of whether RNA editing will become a mainstream platform or remain a niche approach for specific diseases. #RNAEditing #Biotech #AscidianTherapeutics #StargardtDisease #GeneTherapy #FDA #DrugDevelopment #Business #Startup #LifeSciences #CRISPR #BaseEditing #ABCA4 #ClinicalTrials #Neuroscience #Ophthalmology #FexingoBusiness #BusinessPodcast Keep every episode free: buymeacoffee.com/fexingo