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

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


Episode 36


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 Tolerance

Uduak Thomas, GEN, October 6, 2025


Metal-Organic Frameworks Win the 2025 Nobel Prize in Chemistry

Julianna LeMieux, PhD, GEN, October 8, 2025


Gene Therapy Significantly Slows Huntington Disease Progression

GEN, September 24, 2025

 

StockWatch: uniQure Shares Reach Five-Year High on “Game Changing” Huntington’s Data

Alex Philippidis, GEN Edge, September 28, 2025

 

Chiesi, Arbor Target Rare Liver Diseases in Up-to-$2.1B Gene Editing Collaboration

Alex Philippidis, GEN Edge, October 8, 2025


Touching Base Podcast

Hosted by Corinna Singleman, PhD


Hosted on Acast. See acast.com/privacy for more information.


Published on 14 hours ago






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