
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 Notes0:00 Intro to The Genetics Podcast01:27 Welcome to Salvador and how he became involved in drug development11:29 Frustrations and rewards of the genetics field14:27 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 them24:50 Encoded Therapeutics‘ approach to drug discovery and what motivated Salvador to join the team27:50 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 psychiatry37:03 Considerations for using natural history studies41:00 Expectations and goals for Encoded Therapeutics’ current and upcoming studies43:45 Closing remarksFind out moreEncoded TherapeuticsNew clinical data
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EP 242: Connecting dementia research, policy, and patient communities with Angela Bradshaw of Alzheimer Europe [Re-Run]

EP 240: Hijacking DNA repair machinery to treat Huntington’s disease with Vincent Dion of the UKDRI

EP 239: What long-read sequencing reveals about Alzheimer’s and ALS with Paul Valdmanis of the University of Washington

EP 238: Uncovering epistatic interactions in complex disease with machine learning with Bin Yu of UC Berkeley
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