EP 209: Reinventing Fabry disease treatment with Chris Hopkins of Glafabra Therapeutics

This week on The Genetics Podcast, Patrick is joined by Chris Hopkins, CEO of Glafabra Therapeutics. They discuss how Glafabra is advancing a next-generation cell therapy for Fabry disease, the differences between gene therapy modalities, and the future of cell and gene therapy innovation.

Show Notes: 

0:00 Intro to The Genetics Podcast

00:59 Welcome to Chris

01:28 Overview of Fabry disease and reviving a promising shelved gene therapy

03:12 Limitations of current Fabry disease treatments versus Glafabra Therapeutics’ approach

05:19 How autologous cell therapy avoids an immune response

06:43 Comparing this cell therapy approach to that of Casgevy for sickle cell disease

11:28 Expanding Glafabra’s platform to other lysosomal storage disorders through enzyme cross-correction

13:47 Comparing autologous cell therapy and AAV in Fabry

17:02 Path to clinical development and funding strategy for Glafabra’s lead program

19:33 Cost efficiency and trial design advantages of an orphan drug approach

21:19 Considerations around comparator groups for Glafabra’s therapy

24:11 Underdiagnosis and hidden prevalence of rare diseases

25:53 Other innovations Chris is excited about and expectations for the future of cell and gene therapy

31:56 How Chris invented a technique to safely “milk” venomous cone snails

37:00 Closing remarks and advice for scientists taking the leap from academia to entrepreneurship

Find out more

• Glafabra Therapeutics (https://www.glafabra.com/)

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