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The Genetics Podcast
Sano Genetics
207 episodes
1 day ago
Exploring all things genetics. Dr Patrick Short, University of Cambridge alumnus and CEO of Sano Genetics, analyses the science, interviews the experts, and discusses the latest findings and breakthroughs in genetic research. To find out more about Sano Genetics and its mission to accelerate the future of precision medicine visit: www.sanogenetics.com
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Natural Sciences
Science
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All content for The Genetics Podcast is the property of Sano Genetics and is served directly from their servers with no modification, redirects, or rehosting. The podcast is not affiliated with or endorsed by Podjoint in any way.
Exploring all things genetics. Dr Patrick Short, University of Cambridge alumnus and CEO of Sano Genetics, analyses the science, interviews the experts, and discusses the latest findings and breakthroughs in genetic research. To find out more about Sano Genetics and its mission to accelerate the future of precision medicine visit: www.sanogenetics.com
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Natural Sciences
Science
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EP 205: From father to biotech founder: Building hope for children with ultra-rare diseases with Terry Pirovolakis of Elpida Therapeutics
The Genetics Podcast
43 minutes 46 seconds
2 weeks ago
EP 205: From father to biotech founder: Building hope for children with ultra-rare diseases with Terry Pirovolakis of Elpida Therapeutics

This week on The Genetics Podcast, Patrick is joined by Terry Pirovolakis, CEO and Founder of Elpida Therapeutics. They discuss Terry’s journey to create a life-saving gene therapy for his son, the founding of Elpida Therapeutics to bring hope to families with ultra-rare diseases, and the challenges of scaling therapies that aren’t commercially viable.

Show Notes: 

0:00 Intro to The Genetics Podcast

00:58 Welcome to Terry

01:24 The beginning of Terry’s gene therapy journey after his son Michael was diagnosed with SPG50

03:24 Learning the biotech industry and building a gene therapy team

05:17 Terry’s experience with learning about gene therapy without a scientific background

06:42 The process of building a gene therapy in under three years during the COVID-19 pandemic

09:30 Fundraising through community support and major donors

11:06 Expanding access of Michael’s gene therapy to children all over the world

12:32 The creation of Elpida Therapeutics to develop non-commercially viable therapies and adapting to a challenging funding landscape

15:24 Insight into cost, accessibility, and the role of endpoints and manufacturing

20:06 Learning from safety events as gene therapy scales and the importance of considering the risk/reward ratio in rare disease

23:24 Landscape of precision therapeutics available today beyond AAV vectors

27:09 Designing trials at Elpida to demonstrate efficacy in ultra-rare disease

29:24 Adapting meaningful endpoints to disease progression and FDA flexibility 

34:02 Background of Priority Review Vouchers and the negative impact of its non-renewal on rare disease funding

37:48 Finding optimism in rare disease family initiatives and gene therapy successes, and the future of advanced therapeutics 

41:07 How to support the rare disease community and families 

44:16 Closing remarks

Find out more

  • Elpida Therapeutics (https://www.elpidatx.com/)

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The Genetics Podcast
Exploring all things genetics. Dr Patrick Short, University of Cambridge alumnus and CEO of Sano Genetics, analyses the science, interviews the experts, and discusses the latest findings and breakthroughs in genetic research. To find out more about Sano Genetics and its mission to accelerate the future of precision medicine visit: www.sanogenetics.com