EP 195: From junk to therapy: The untapped power of regulatory RNAs with David Bumcrot of CAMP4 Therapeutics

Summary: This week on The Genetics Podcast, Patrick is joined by David Bumcrot, Chief Scientific Officer of CAMP4 Therapeutics. They discuss David’s involvement in the development of the breakthrough technologies in RNA interference and CRISPR, how CAMP4 is pioneering the use of regulatory RNAs to treat haploinsufficient diseases, and the complexities of gene regulation. Show Notes: 0:00 Intro to The Genetics Podcast00:59 Welcome to David 01:57 David’s experiences at companies that developed RNA interference (RNAi) and CRISPR technologies04:05 Lessons from translating RNAi to the bedside 05:58 The scalable chemistry of RNA versus the complexity of gene editing07:48 Insight into the lack of rapid scalability and growth in gene therapy companies09:19 How CAMP4 is using regulatory RNAs to boost gene expression in haploinsufficient diseases13:01 Mapping and predicting the impact of specific regulatory RNAs on the expression of target genes15:05 Why regulatory RNAs open up new target space without needing new drug modalities16:27 How CAMP4 prioritizes certain diseases based on delivery feasibility and genetic evidence18:31 Understanding expression thresholds in haploinsufficiency and building confidence in therapeutic targets 20:29 Comparing different approaches for increasing gene expression22:39 Other therapeutic areas of focus for CAMP4, including urea cycle disorders and central nervous system (CNS) disorders25:47 Lessons from early clinical development and engaging regulators on novel targets27:47 Building high-resolution regulatory RNA maps beyond what public datasets can provide31:04 Considerations around the treatment window for SYNGAP132:40 The value of collaborative frameworks for rapid therapy development 35:20 Rethinking early safety assessment and global regulatory strategies for RNA therapies38:37 The importance of basic science, staying optimistic, and continuing to invest in biotech innovation40:18 Closing remarksFind out moreCAMP4 Therapeutics (https://www.camp4tx.com)Please consider rating and reviewing us on your chosen podcast listening platform! https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link