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)
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
Popular Podcasts
My Favorite Murder with Karen Kilgariff and Georgia Hardstark
My Favorite Murder is a true crime comedy podcast hosted by Karen Kilgariff and Georgia Hardstark. Each week, Karen and Georgia share compelling true crimes and hometown stories from friends and listeners. Since MFM launched in January of 2016, Karen and Georgia have shared their lifelong interest in true crime and have covered stories of infamous serial killers like the Night Stalker, mysterious cold cases, captivating cults, incredible survivor stories and important events from history like the Tulsa race massacre of 1921. My Favorite Murder is part of the Exactly Right podcast network that provides a platform for bold, creative voices to bring to life provocative, entertaining and relatable stories for audiences everywhere. The Exactly Right roster of podcasts covers a variety of topics including historic true crime, comedic interviews and news, science, pop culture and more. Podcasts on the network include Buried Bones with Kate Winkler Dawson and Paul Holes, That's Messed Up: An SVU Podcast, This Podcast Will Kill You, Bananas and more.
24/7 News: The Latest
The latest news in 4 minutes updated every hour, every day.
Dateline NBC
Current and classic episodes, featuring compelling true-crime mysteries, powerful documentaries and in-depth investigations. Follow now to get the latest episodes of Dateline NBC completely free, or subscribe to Dateline Premium for ad-free listening and exclusive bonus content: DatelinePremium.com