Summary: 

This week on The Genetics Podcast, Patrick is joined by Frank Gentile, Chief Executive Officer of Casma Therapeutics. They discuss the role of autophagy in neurodegenerative diseases, Casma’s work on therapies for Gaucher’s disease and Parkinson’s disease, and the challenges and opportunities in rare disease biotech.

Show Notes: 

0:00 Intro to The Genetics Podcast

00:58 Welcome to Frank 

02:00 Origin story for Casma Therapeutics and its therapeutic focus on autophagy

04:51 Diseases in which autophagy is dysregulated

07:46 The link between GBA1 and Parkinson’s, and why so many neurodegenerative diseases exhibit autophagy defects

12:28 Findings from Casma Therapeutics’ preclinical studies and associated mechanisms

16:46 Milestones and design of Casma Therapeutics’ upcoming clinical studies

20:24 Well-characterized cohorts of GDPD patients from natural history studies

21:55 Identifying alternative therapeutic targets involved in autophagy initiation that are mTOR independent

24:46 The negative effects of broad inhibition of mTOR

25:57 Advantages and disadvantages of using small molecule therapy versus broader gene therapy

26:59 Frank’s experience in investment and his approach to risk

29:15 Frank’s perspective on the current biotech climate and how investors view rare disease

32:16 Extending lead candidate and portfolio strategy to other therapeutic applications 

34:15 Partnering opportunities and future potential of the TRPML1 program across neurodegenerative diseases

35:50 A potential link between autophagy pathways and longevity

37:20 Closing remarks

Find out more

• Casma Therapeutics (https://www.casmatx.com/)

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