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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