Ep. 18: Speed vs. Safety: Rapid Approvals from the FDA

This month I am joined by Benjamin Oakes, scientist, entrepreneur, and co-founder of Scribe Therapeutics. He studied at UC Berkeley, working in the Doudna Lab and Savage Lab during the earliest days of CRISPR. We discuss his personal vision for the future of CRISPR, encouraging advances in the field, and the first ever drug approval for a CRISPR based therapy. 

Norm Wainwright and Foster Jordan have been immersed in the field of limulus amebocyte lysate (LAL) testing for decades, and have seen the progress and refinement of this crucial safety test. With the 20th anniversary of Charles River's Endosafe Cartridge and the recent introduction of recombinant alternatives, we look back at the past and future of endotoxin testing.

I am joined by four experts in their field to discuss what will be hot in rare diseases, drug discovery, artificial intelligence, and animal models in 2024. 

Molly Dupre, a lab assistant at Charles River's Reno site, was recently diagnosed with Niemann-Pick Type B/ Acid Sphingomyelinase Deficiency. After years of waiting, doctor's visits, and tests, she could finally put a name to the condition that flared up after the birth of her son. But what came next? She joins me to tell her story.

Senior Principal Scientific Advisor Stan Spence joins me to discuss off-target liability: what happens when a drug misses its target? What are the risks to the patient if the off-target effects aren't caught early? Has there ever been an example of an off-target effect being beneficial to the patient? Find out here!

Rachel Kiserow and Susan Desmond have joined us year after year as part of Eureka's Breast Cancer Roundtable. This year, they wanted to share what happens after - after the treatments, after remission - life after cancer.

For this episode, I am joined by Charles River's Doug Botkin and Pure Microbiology's Abby Roth to discuss the 2012 NECC meningitis outbreak. Doug and Abby bring their background in microbiology to explain how it happened, and how we can prevent such tragedies in the future. 

Charles River experts Justin Bryans and Elizabeth Anderson join me for a spirited discussion of where we are now in oncology research. Could we ever see a world without cancer?

Manufacturing gene therapies is a hot topic in the pharma field, on both the supplier and the client ends. Joining me to discuss this issue is Charles River Associate Director of Gene Therapy, Andrew Frazer, and Purespring Therapeutics' VP of Chemistry, Manufacturing and Controls, Amanda Weiss. 

Dr. Lauren Black and Dr. Monkol Lek are both committed to the research and advancement of rare disease treatments. But when you are trying to treat just one patient, what can you learn that could help many more? We discuss the importance of n-of-1 studies, not just for that one patient and their families, but for anyone who might someday need a rare cure.