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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