EP 241: The hard-won lessons behind Encoded Therapeutics’ Dravet syndrome gene therapy with Salvador Rico [Re-run]

This week on The Genetics Podcast, Patrick is joined by Salvador Rico, Chief Medical Officer at Encoded Therapeutics. With Encoded’s Dravet syndrome gene therapy program recently reaching major clinical and regulatory milestones, we’re revisiting this conversation on Salvador’s journey into drug development, his work on gene therapy for X-linked myotubular myopathy, and the fundamental challenges and exciting advances shaping the future of genetic medicine.

Show Notes

0:00 Intro to The Genetics Podcast

01:27 Welcome to Salvador and how he became involved in drug development

11:29 Frustrations and rewards of the genetics field

14:27 Salvador’s study on gene therapy for patients with X-linked myotubular myopathy (XLMTM)

20:13 Risk of liver issues in gene therapy trials and attempts to mitigate them

24:50 Encoded Therapeutics‘ approach to drug discovery and what motivated Salvador to join the team

27:50 Steps towards therapeutic targeting of gene regulatory elements 

30:32 Advantages of different methods for drug delivery 

32:59 DNA- versus RNA-based therapy

35:24 Insights from approaches in other fields, including psychiatry

37:03 Considerations for using natural history studies

41:00 Expectations and goals for Encoded Therapeutics’ current and upcoming studies

43:45 Closing remarks

Find out more

• Encoded Therapeutics

• New clinical data