Piotr Kosla, founder of the PACS2 Research Foundation, joins us to discuss his personal motivation to develop the first treatment for Development and Epileptic Encephalopathy 66 (PACS2 Syndrome), how cell painting assays are being used to repurpose a treatment for this condition, what the future holds for research in this area, and how you can help contribute to his mission.

Show Notes

• PACS2 Research Foundation
• Eureka Blog | Drug Repurposing Through Cell Painting Could Treat Rare Disease
• ASO Screening Services
• Gene Therapy Services
• Art of Science: Cell Painting
• Free for Life: The Story of n-Lorem Foundation

Lisa Sharp, Project Coordinator for Charles River, joins us to discuss her COVID lockdown experience. Right at the beginning of the pandemic, just weeks apart, both she and her husband were diagnosed with cancer. Sadly, he didn't make it. While Lisa and their daughter faced the worst, she decided to make big changes to her life and career. Listen now to hear her story.

Imagine you’re exploring the great outdoors when, out of nowhere, you feel the fangs of a snake – a poisonous snake – breaking through your skin. Medical care is nowhere in sight, so you’re left with the terrifying prospect of venom slowly infecting your body’s critical functions.

This is the reality that roughly five million people face annually. Over 500,000 of which either die or are permanently maimed or suffer long-term cardiovascular/neurological impairments.

However, Drs. Rebecca Carter and Matt Lewin are working to change that. Their work at Ophirex revolves around further developing Varespladib, a repurposed drug, as an accessible and affordable oral treatment to instantly combat venomous snakebites. By ingesting a small tablet or capsule, victims can buy time to seek out more advanced care, giving them a better chance to limit complications and prevent death.

Join us as we explore the impacts snake venom has on the body, how Drs. Carter and Lewin discovered Varespladib, how collaborations and partnerships have benefited their development of this treatment, and what can be done to improve its accessibility, especially in remote areas of the world.

Show Notes

Ophirex Lifesaving Antidotes

Antivenoms -_World Health Organization

Interventions for the Preventing Reactions to Snake Venoms - National Institute of Health

I am joined by Dr. Kacey Ronaldson-Bouchard from Columbia University, who is an expert on linking organs on a chip together to form models of human systems. Can these models replace animals? Can they save time and money in drug development and safety testing? Find out now when you listen to this podcast!

Mike Curtis, president and CEO of eGenesis, and transplant surgeon Dr. Leonardo Riella join me to discuss the ongoing research into pig kidney to human transplants. How can a porcine kidney be genetically modified to be safe for a human? Listen now to find out more.

Felicia Samuel has worked for Charles River for years, but recently the work she contributes to has taken on a new meaning. Her daughter will be part of a clinical trial to receive one of the brand new sickle cell treatments, with the potential to drastically change her own and her family's lives. She joins me to discuss the details of this amazing but involved gene therapy process, and how working for one of the companies that contributed to this treatment's research makes her feel. You can read more about it here: https://www.criver.com/eureka/crispr-silences-sickle-cell

What if we could replace mouse model control groups with a virtual equivalent? What would that mean for drug development in terms of cost, time, and animal model reduction? To answer, I am joined by Charles River associate director Laura Lotfi and Guillemette Duchateau-Nguyen from F. Hoffmann-La Roche Ltd, Pharmaceutical Sciences - Roche Innovation Center Basel. Listen now to learn how close we are to replacing some physical models with 1's and 0's.

Alex Sargent, Director of Process Development at Charles River in our Cell and Gene Therapy department, joins me to discuss the work he and his partner put into developing an algorithm to make his job faster and easier. How can you train a computer to accurately sift through the mountains of data generated in drug development? Listen now to find out!