EP 208: Gene therapy at a crossroads: Successes, concerns, and the path forward скачать в хорошем качестве

EP 208: Gene therapy at a crossroads: Successes, concerns, and the path forward

This week on The Genetics Podcast, Patrick Short takes a solo deep dive into the current state of gene therapy ahead of next week’s live recording at ASHG 2025. He explores the promise and limitations of adeno-associated virus (AAV) delivery, examples of gene therapies for neuromuscular diseases, and the challenges of balancing safety, cost, and commercial viability in rare diseases. Show Notes: 0:00 Intro to The Genetics Podcast 00:59 Episode overview 02:50 Definition and scope of gene therapy 04:50 Gene therapy delivery via adeno-associated virus (AAV) and associated challenges and advantages 06:40 AAV-based gene therapy and other advanced therapies in spinal muscular atrophy (SMA) 10:29 Recent safety concerns around Sarepta Therapeutics’ AAV-based gene therapy for Duchenne muscular dystrophy (DMD) 19:30 Commercial viability challenges for rare disease gene therapy 24:26 Risk-benefit analysis of gene therapy for rare diseases 28:33 Considerations for optimizing AAV design and delivery routes 31:26 Alternative approaches for delivery using viral and non-viral methods 36:09 The future of AAV gene therapy 41:42 Closing remarks Find out more Update on Sarepta Therapeutics’ gene therapy for DMD (https://www.fiercepharma.com/pharma/s...) #TheGeneticsPodcast #Genetics #PrecisionMedicine #GeneTherapy #RareDisease #AAV