Vital Science

Drug developers face long odds and complex regulatory challenges when it comes to crafting a suitable candidate. In fact, only one of every 10 drug candidates gain regulatory approval, and when they do, it takes an average of 14 years and costs well over $2 billion.   So what makes for a successful candidate? It starts with a suitable screening platform that identifies targets and the molecules that bind to them to create a therapeutic response. Retrogenix is one such platform that the top 20 drug developers in the world currently use to increase their chances of success thanks to its unique technology that doesn’t require animal models or human tissue to evaluate effectiveness.   Join us to find out how Retrogenix works to identify targets, accelerate drug development, get vulnerable patients the therapies they need more quickly, and adapt to meet the growing demands of regulatory agencies. Show Notes  Charles River Acquires Retrogenix  Retrogenix Cell Microarray Technology  De-risking Therapies and Vacci

Aled Edwards’ vision of science is set to shake the foundation of drug discovery and development as we know it.   The Director of M4K Pharma and CEO of the Structural Genomics Consortium is leading the charge for a more open scientific approach. It’s a parallel universe where scientists can work together and get therapies designed to treat rare diseases in the hands of patients even faster – one that isn’t bound by patents, siloes, and profits.  Join Aled as he discusses the “open science” approach, the current state of the pharmaceutical industry, and what the future holds for genetic biology.Show Notes  M4K Pharma Open Science for Children's Health  Structural Genomics Consortium  The Disruptors - Meet Aled  A Deadly Brain Cancer in Children  Tackling DIPG with Open Science  The Journal of Medicinal Chemistry—Leveraging an Open Science Drug Discovery Model to Develop CNS-Penetrant ALK2 Inhibitors for the Treatment of Diffuse Intrinsic Pontine Glioma  Resources  M4K Pharma Open Science for Children's H

A medic by trade, Mark Kotter has always held a particular interest in neuroscience and stem cell research.As founder and CEO of bit.bio, he's overseen the development of a unique approach to "reprogram" stem cells and change them into consistent and scalable disease-relevant cells, which can then be screened for potential therapeutics to help treat such diseases as Huntington's Alzheimer's, and ALS.Hear about this revolutionary technology and how it works, what benefits it can provide researchers, and how it could very well shape the future of drug development.Show Notes  Stem Cells for Drug Discovery  Building a Stable of Stem Cells  Conversations in Science With Disruptors Leading the Way  Neuroscience | Charles River  bit.bio  Resources  https://www.bit.bio/news/focus-on-a-founder-with-mark-kotter-celebrating-10-years-of-community  https://www.criver.com/eureka/podcast/e28-building-the-building-blocks-of-life   https://www.criver.com/about-us/disruptors/meet-mark?region=3601  https://www.bit.bio/

Imagine a gene therapy treatment that gives drug-resistant focal epilepsy patients more control over their seizures and ultimately improve their quality of life?We sat down with Karin Agerman to discuss this possibility and discovered that her work at Combigene is at the forefront of a novel gene therapy called CG01.Find out how this single-injection therapy is giving renewed hope to this patient population and learn more about CombiGene’s research into CG01.SHOW NOTES Cell and Gene Therapy Services and Products CombiGene Gene Therapy, A Promising Future for Epileptic Drug Development High Quality Plasmid DNA: A Case for Phase Dependent Approaches to Manufacturing Tailored Preclinical Support for Your Cell & Gene Therapies Digital Resources Cell and Gene Therapy Development Video Series Charles River Completes the Acquisition of Cognate BioServices CombiGene initiates GMP production of CG01 for the first-in-human study Navigating the ATMP Regulatory Landscape

In March 2020, we invited Josh Cohen and Justin Klee on Vital Science to discuss Amylyx Pharmaceuticals, a company that had one simple mission – to improve the quality of life for those battling neurodegenerative diseases.   Since their humble beginnings, what was once a company with less than ten employees has grown exponentially. Get the latest from Josh and Justin as they rejoin our podcast to discuss the new drug application process for AMX0035, what they’ve learned from their experiences, and what lies ahead.  See what Amylyx Pharmaceuticals is currently working on and get updates on clinical trials.

The role of human cells and tissues is key in the development of next-generation therapies. With just a few samples, donors contribute to scientific advancements in medicine. This is especially true with cell and gene therapy, a field that is projected to grow over the next several years, and with it comes the growing need to find reliable and recallable donors willing to help those in need. In our latest episode of Vital Science, learn more about the critical role these unsung heroes play in advancing the future of therapeutic development. Show Notes  The Link Between a Consistent Cell Supply and Successful Cell Therapies  A Reliable Donor Network is the Foundation for Development of Successful Cell-Based Therapies  What Impacts Starting Material Quality? Part 1: Donor Variability  Developing Cell Therapies? Donors Matter.  Resources  Hemacare  Cell and Gene Therapy Services and Products 

From a young age, Kim Noonan knew what she wanted in life — a career in science and a desire to help.When her mother succumbed to gastric cancer, it drove her even more to help people, especially cancer patients. Today, she’s at the forefront of a potentially game-changing development in cancer therapy — using bone marrow-infiltrating lymphocytes (MILs) designed to target and kill cancerous tumors of all shapes and sizes.Find out how her early work with myeloma patients started her road to discovering MILs, the origin of WindMIL Therapeutics, challenges associated with manufacturing MIL-based therapies, and what the future holds for this revolutionary treatment.Learn more about WindMIL Therapeutics and the science behind their work with bone marrow immunology.SHOW NOTES Cell and Gene Therapy Services Oncology Research and Drug Development The Link Between a Consistent Cell Supply and Successful Cell Therapies Charles River Presents: Tailored Preclinical Support for Your Cell & Gene Therapies Cell and Gene

Huntington’s disease may only affect one in every 10,000 people in the United States, but its symptoms can drastically debilitate a person’s quality of life.For Ignacio Muñoz-Sanjuan, he witnessed first-hand the devastating effects this disease can have. He’s visited parts of the world that have the highest prevalence of Huntington’s disease on the planet, including Taiwan and South America. Today, he’s involved with two different nonprofit organizations seeking to accelerate the development of treatments to help those afflicted by, or at risk for, this fatal condition.Join us as we explore Ignacio Muñoz-Sanjuan’s journey in raising awareness about Huntington’s disease, how Charles River contributed to developing a framework for potential treatments, and what he believes the future holds for drug development in this disease area.Discover how the CHDI Foundation and Factor-H are working to help those suffering from Huntington’s disease and how you can get involved. Huntington’s Disease (HD) Studies  DNA Dama

Sanath Ramesh was determined to track down the resources needed to repurpose a drug for his son Raghav’s ultra-rare disease. This determination led him to apply his experiences and empower the rare disease community with his collaborative platform, the OpenTreatments Foundation. Find out how his repurposed drug platform is now giving hope to other rare disease patients.EPISODE LINKOTHER RESOURCES:   Open Treatments  Repurposing Compound Libraries  High-Throughput Screening 

What if it was possible to speed up drug development without having to reinvent the wheel? By repurposing existing drugs, drug hunters are taking a giant step forward towards changing the future of drug discovery. Join our panel of drug hunters to hear how they reinvent previously approved drugs to get a head start towards treating patients. Acknowledgements: Hosted by: Chris GarciaNarrated by: Gina Mullane and Chris Garcia  Special thanks to:  Ian WaddellVad LazariBarbara Killian

What if the cure for your untreatable disease was just waiting on the shelf at your neighborhood pharmacy? Join physician, patient, and researcher Dr. David Fajgenbaum as he shares how his unexpected battle with Castleman disease set him on a journey to find his cure and develop new ways of using existing drugs.  Acknowledgements: Hosted by: Chris GarciaNarrated by: Gina Mullane  Special thanks to:  Dr. David FajgenbaumBarbara Killian

Our panel explores the vital role horseshoe crabs play in bacterial endotoxin testing and the ongoing battle to preserve the population.

How are 3D cell models changing our ability to create more effective therapies? Our panel of scientific experts discuss.

Dr. David Fischer joins our panel to discuss the evolution, application, and potential of CRISPR/Cas9 gene editing technology.

In this brief recap, our hosts Gina Mullane and Chris Garcia discuss topics covered in our series on vaccines.

Dr. Sarah Gould returns to provide an update on the development of a vaccine for COVID-19 and what lies ahead.

Distinguished scientist Dr. Lauren Black discusses a “weight-of-evidence” approach to accelerated vaccine development and approvals.

Dr. Christina Satterwhite delves into the immune system and how vaccines target harmful pathogens. 

Dr. Sarah Gould explains how infectious disease spurred the discovery and subsequent evolution of vaccines.

Two sisters share their experience with clinical trials for cystic fibrosis.