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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