Nobel Prizes Announced and Breakthroughs for Huntington’s and Rare Diseases

Metal-organic frameworks and peripheral immune tolerance were the big winners of the Nobel prizes in chemistry, and in physiology or medicine, respectively. In this episode of the podcast, we discuss the winners and the impacts of their discoveries. Then we move over to some business news where we discuss a novel gene therapy for Huntington’s disease from uniQure that made waves recently. Early clinical trial data for AMT-130 showed that it could meaningfully slow the progression of the disease by as much as 75%. Also in business news, a new partnership involving Arbor Biotechnologies and Chiesi Group aims to develop gene editing therapies to target rare liver diseases. Join GEN editors Corinna Singleman, PhD, Alex Philippidis, and Uduak Thomas for a discussion of the latest biotech and biopharma news. Listed below are links to the GEN stories referenced in this episode of Touching Base:Brunkow, Ramsdell, Sakaguchi Win Nobel Prize in Physiology or Medicine for Peripheral Immune ToleranceUduak Thomas, GEN, October 6, 2025Metal-Organic Frameworks Win the 2025 Nobel Prize in ChemistryJulianna LeMieux, PhD, GEN, October 8, 2025Gene Therapy Significantly Slows Huntington Disease ProgressionGEN, September 24, 2025 StockWatch: uniQure Shares Reach Five-Year High on “Game Changing” Huntington’s DataAlex Philippidis, GEN Edge, September 28, 2025 Chiesi, Arbor Target Rare Liver Diseases in Up-to-$2.1B Gene Editing CollaborationAlex Philippidis, GEN Edge, October 8, 2025Touching Base PodcastHosted by Corinna Singleman, PhD Hosted on Acast. See acast.com/privacy for more information.