Summary: 

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. 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/sarepta-shares-more-elevidys-safety-data-response-patient-group-fda-petition)

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