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 Podcast
00:59 Welcome to David
01:57 David’s experiences at companies that developed RNA interference (RNAi) and CRISPR technologies
04:05 Lessons from translating RNAi to the bedside
05:58 The scalable chemistry of RNA versus the complexity of gene editing
07:48 Insight into the lack of rapid scalability and growth in gene therapy companies
09:19 How CAMP4 is using regulatory RNAs to boost gene expression in haploinsufficient diseases
13:01 Mapping and predicting the impact of specific regulatory RNAs on the expression of target genes
15:05 Why regulatory RNAs open up new target space without needing new drug modalities
16:27 How CAMP4 prioritizes certain diseases based on delivery feasibility and genetic evidence
18:31 Understanding expression thresholds in haploinsufficiency and building confidence in therapeutic targets
20:29 Comparing different approaches for increasing gene expression
22:39 Other therapeutic areas of focus for CAMP4, including urea cycle disorders and central nervous system (CNS) disorders
25:47 Lessons from early clinical development and engaging regulators on novel targets
27:47 Building high-resolution regulatory RNA maps beyond what public datasets can provide
31:04 Considerations around the treatment window for SYNGAP1
32:40 The value of collaborative frameworks for rapid therapy development
35:20 Rethinking early safety assessment and global regulatory strategies for RNA therapies
38:37 The importance of basic science, staying optimistic, and continuing to invest in biotech innovation
40:18 Closing remarks
Find out more
CAMP4 Therapeutics (https://www.camp4tx.com)
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