This week on The Genetics Podcast, Patrick is joined by Richard Wilson, Senior Vice President, Primary Focus Lead of Genetic Regulation at Astellas. They discuss where the gene therapy field stands today, diving into successes, persistent barriers, regulatory considerations, manufacturing strategies, and other reflections on important approaches to precision medicine drug development.

Show Notes: 

0:00 Intro to The Genetics Podcast

00:59 Welcome to Richard

01:33 Current wins and challenges in gene therapy 

07:13 The need for more open non-competitive data sharing and a clearer pathway towards reimbursement and incentivization

11:15 Building post-approval systems into the genomic medicine lifecycle

14:47 Rethinking evidence standards in rare disease trials, and the global push for regulatory and reimbursement alignment

20:28 Genetic medicine breakthroughs in central nervous system diseases

22:25 The challenges of starting clinical development with an end in mind

24:34 The need for careful analysis around endpoints, vector design, and delivery approaches

29:33 Navigating regulatory hurdles when making mid-program changes to vectors or payloads

31:16 Strategies for enhancing scalability and quality of gene therapy manufacturing 

36:05 Exploring other delivery methods beyond AAV

39:32 Getting ready for the Timmerman Traverse and raising nearly $1M for Life Science Cares

43:48 A call to refocus on patients, partnership, and purpose in uncertain times

45:55 Closing remarks 

Find out more

• Astellas (https://www.astellas.com/)

• Timmerman Traverse fundraising for Life Science Cares (https://timmermantraverse.blackbaud-sites.com/)

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