logo
episode-header-image
Jul 10
41m 32s

EP 195: From junk to therapy: The untapp...

Sano Genetics
About this episode

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

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


Up next
Jul 3
EP 194: Ontologies, diagnostics, and genomics for all with Melissa Haendel of UNC Chapel Hill
Summary: This week on The Genetics Podcast, Patrick is joined by Melissa Haendel, Director of Translational Informatics and Precision Health and Professor at the University of North Carolina at Chapel Hill, and Phenotypic Lead at Alamya Health. They discuss the limitations of ele ... Show More
42m 51s
Jun 26
EP 193: Targeting autophagy to treat neurodegeneration with Frank Gentile of Casma Therapeutics
Summary: This week on The Genetics Podcast, Patrick is joined by Frank Gentile, Chief Executive Officer of Casma Therapeutics. They discuss the role of autophagy in neurodegenerative diseases, Casma’s work on therapies for Gaucher’s disease and Parkinson’s disease, and the challe ... Show More
38m 42s
Jun 19
EP 192: From $88 to breakthroughs: Innovation in retinal gene therapy with Dr. Huma Qamar of Ocugen
Summary: This week on The Genetics Podcast, Patrick is joined by Huma Qamar, Chief Medical Officer at Ocugen. They discuss her journey from arriving in the US with $88 in her pocket to leading gene therapy programs, Ocugen’s breakthrough in retinal disease, and how gene-agnostic ... Show More
34m 59s
Recommended Episodes
Apr 2024
PT500 – Shulgin Farm and the Future of Psychedelic Drug Development, with Paul F. Daley, Ph.D.
In this episode, Joe interviews Paul F. Daley, Ph.D., who worked with Sasha Shulgin in his lab for the last seven years of his life, helping him finish (and co-authoring) "The Shulgin Index, Volume One: Psychedelic Phenethylamines and Related Compounds." He is now the co-founder, ... Show More
36m 1s
Mar 2025
The Science, Evolution, and Future of Lifespan and Healthspan with Dr. Michael Ringel
On this episode of Longevity by Design, Dr. Gil Blander speaks with Dr. Michael Ringel, COO of Life Biosciences, about the science of aging and longevity. Michael discusses how evolution influences lifespan and why aging occurs. He explains the trade-off between resource allocati ... Show More
1h 12m
Jan 2025
Rachel Chevalier - Update on Topical Steroids for EoE
In this episode, hosts Drs. Temara Hajjat and Jenn Lee talk to Dr. Rachel Chevalier. Dr. Chevalier is a pediatric gastroenterologist and physician scientist at Children's Mecy Hospital. She is an Associate Professor at the University of Missouri-Kansas City School of Medicin ... Show More
54m 42s
Jul 2024
Exploring Pain Science with Paul Ingraham: From Massage Therapy to Scientific Writing
In this episode, we welcome Paul, a former massage therapist turned scientific writer, who shares his journey from clinical practice to founding the PainScience.com website. Paul discusses the evolution of his career, the challenges of transitioning to science writing, and the im ... Show More
52m 6s
May 5
#347 – Peter’s takeaways on mastering sleep, dealing with chronic pain, developing breakthrough cancer drugs, transforming healthcare with AI, advancing radiation therapy, and healing trauma | Quarterly Podcast Summary #5
View the Show Notes Page for This Episode Become a Member to Receive Exclusive Content Sign Up to Receive Peter’s Weekly Newsletter In this quarterly podcast summary (QPS) episode, Peter summarizes his biggest takeaways from the last three months of guest interviews on the podcas ... Show More
33m 20s
Aug 2024
Enhancing Muscle Quality and Longevity | Dr. Anurag Singh
Discover the groundbreaking science behind healthy aging in this episode of the Dr. Gabrielle Lyon Show! Join Dr. Lyon as she sits down with Dr. Anurag Singh, an MD PhD with extensive research in immunology, to explore the incredible benefits of Urolithin A, a compound that's sho ... Show More
1h 31m
Mar 2024
Episode 19: Extracorporeal Blood Purification in the Context of Multiorgan Support: Current Role and Best Practices
Episode 19 is the third episode in KDIGO's three-episode series on acute kidney injury (AKI), hosted by Dr. Kathleen Liu (University of California at San Francisco, United States). In this episode, Dr. Liu and Dr. Claudio Ronco (International Renal Research Institute of San Borto ... Show More
11 m
Dec 2024
Cellular Health - Adeel Khan, MD - Regenerative Medicine
We’re thrilled to announce our latest podcast episode featuring Dr. Adeel Khan, a trailblazer in regenerative medicine. Join Dr. Greg Kelly and Dr. Khan as they explore groundbreaking approaches to healthcare that focus on repairing and regenerating tissues at a cellular level. D ... Show More
46m 41s
Apr 28
#346 - Scaling biotech and improving global health: lessons from an extraordinary career in medicine | Susan Desmond-Hellmann, M.D., M.P.H.
View the Show Notes Page for This Episode Become a Member to Receive Exclusive Content Sign Up to Receive Peter’s Weekly Newsletter Susan Desmond-Hellmann is a physician and scientist whose remarkable career has spanned clinical medicine, oncology, biotech innovation, and global ... Show More
2h 5m