Vital Science: Recent Episodes

Charles River

Life science is complicated, but its mission is simple – to create a safer, healthier future. This podcast tells the stories behind the science from the perspective of patients, scientists, employees, and advocates that have felt its impact.

For decades, researchers have utilized animal models to help predict the effects of a drug compound in humans.

With recent advances in technology and the passage of the FDA Modernization Act 2.0, researchers are increasingly looking at new methods to refine, reduce, and replace in vivo testing to advance drug candidates. However, one important question remains – how are all these modern advancements and laws driving progress in drug discovery?

Find out as Michael Templin, a member of Charles River’s Scientific Advisory Services team, joins us for a look at the impacts of this legislation on regulatory submission for biosimilars, how improving animal welfare will make drug development better, and what knowledge a CRO can bring to optimize projects.

Show Notes

Animals in Research | Charles River
Basic Research | Charles River
Research Models and Services
FDA Modernization Act

Spinocerebellar ataxia type 3 (SCA3) is a neuromuscular disease affecting just 50,000 people in the United States, including three generations of the Klassen family.

Greg Klassen, his father, and his son have all been diagnosed with this rare condition that impacts the central nervous system, affecting balance, coordination, and mood. Motivated by the 50/50 chance his son Jeff will develop its unforgiving symptoms, Greg joined the board of the Cure Rare Disease foundation and is currently in touch with the efforts being made to develop a treatment that will improve his quality of life and others affected by SCA3.

Join us for a conversation on how Greg copes with SCA3 in his own life, what the rare disease community is doing to raise awareness, how antisense oligonucleotides (ASOs) could lead to a potential treatment, and what it would mean to him if a cure was developed.

Show Notes

Charles River | Gene Therapy Services
Safety Assessment with Antisense
Charles Rive | Rare Disease
Rare Diseases - Spinocerebellar Ataxia 3
Cure Rare Disease

Spastic Paraplegia 50 (SPG50) is a neurodegenerative and neurodevelopmental disorder that is known to affect only 80 people around the world, including just one person in Canada.

Unfortunately for Terry Pirovolakis, it happens to be his son, Michael. As a result, he’s teamed up with gene therapy experts, CROs, and nonprofits to research, manufacture, and deliver a therapy to help his son and others suffering from this ultra-rare disease, but the clock is ticking.

Join us as Terry discusses his grassroots efforts to raise money and found the Cure SPG50 Foundation, how SPG50 affects the body, the future of research and development for this disease, and what you can do to help Terry’s efforts.

Show Notes:

Sounds of Science E33: A Father's Fight
Eureka Blog: The Hunt for a Solution to SPG50
Cure SPG50 - We Did It
Rare Disease
Cell and Gene Therapy Products & Services
Neuroscience Studies
Family of baby boy with ultra-rare disease aims to raise $3M for gene therapy
Toronto boy with rare genetic disorder begins treatment after family raises US$3 million
Single-patient gene therapy clinical trial at SickKids carves a path for Precision Child Health
Elpida Therapeutics

For Valerie Estess, finding a cure for ALS is personal.

Her sister, Jenifer, was diagnosed with this crippling disease in 1998, which led to the founding of Project ALS, a nonprofit designed to raise awareness and eventually find a cure. Nearly 25 years later, they have raised over $100 million and helped develop Jacifusen, the first therapy designed to help treat ALS. However, Valerie believes their work is far from finished.

Join us for an intimate discussion on the origins of Project ALS, their collaborative efforts with leading scientists, academic institutions, and contract research organizations to further their knowledge of the disease, and what lies ahead for Project ALS’ mission and research.

SHOW NOTES

Project ALS
The Hermstad Legacy: Advances in Treatments for ALS
Jaci's Genes
Neuroscience Studies
S3, E04: From Humble Beginnings to Potential Treatment for ALS
Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

What happens when you combine an industry-altering, AI-powered platform with a leading CRO's preclinical expertise?

That’s what Valo Health and Charles River are answering with the introduction of Logica™. This revolutionary platform aims to accelerate drug discovery and development by finding the most viable molecules faster, helping biotechs and pharma focus their resources only on the ones that will evolve into therapies that patients will receive more quickly.

Join Guido Lanza (Valo) and Julie Frearson (Charles River) as they discuss the components of Logica™, what makes it unique from other platforms, and how it will transform the future of drug discovery.

Show Notes

https://www.criver.com/products-services/discovery-services/logica-transformative-drug-design
https://www.criver.com/insights/charles-river-and-valo-health-launch-logica
https://www.criver.com/eureka/medicine-meet-ai-how-machine-learning-transforming-drug-discovery
Valo Health Is Partnering With Charles River Laboratories To Accelerate Preclinical Drug Discovery
Charles River, Valo Enter AI-enabled Drug Discovery Pact

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 Vaccines for Use During Pregnancy
Assessing ADC Anticancer Therapeutics via Antibody Specificity Screening
Screening and Profiling Assays
Off-Target Screening Cell Microarray Assay
Webinar: Building a comprehensive lead enabling library
Poster: Off-target Assessment of Biotherapeutics for us During Pregnancy
Case Study: Antibody Drug Conjugate Off-Target Binding Screen

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 Health
SGC
The Disruptors - Meet Aled

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/
Stem Cells for Drug Discovery
Next Generation Reprogramming of Human iPSCs to Generate Disease-specific Glutamatergic Neurons Carrying HTT Gene for Huntington’s Disease Research and Drug Discovery
https://www.criver.com/neuroscience-2021

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

When Chris Claussen’s grandfather succumbed to the crippling effects of Alzheimer’s disease, he knew that it could be coming for him in due time.

From then on, he has made it a “personal crusade” to look at what some may call an unconventional method to improve brain health and overall mood – microdosing psychedelic drugs. By combining first-hand experience with laboratory research, he noticed a correlation between the two, but questions and scrutiny still remain as to its effectiveness and whether psychedelics will ever be accepted by society as a proven method to improve brain function and mental health.

Join us for a deeper dive into the specifics of psychedelic drugs, the concept of microdosing and its role in therapeutic efficacy, and what long-term effects they can have.

Show Notes

Since the recording of this episode, Leiio Wellness has changed its company name to First Person. More information can be found at getfirstperson.com.
Are Psychedelics the Next Approach to Treating Mental Health Disorders?
The Therapeutic Potential of Psychedelic Drugs: Past, Present, and Future
Treatment for Resistant Depression: A multi-scale, systems biology approach

Resources

Are Psychedelics the Next Approach to Treating Mental Health Disorders?
The Therapeutic Potential of Psychedelic Drugs: Past, Present, and Future
Treatment for resistant depression: A multi-scale, systems biology approach

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 Therapy Development Video Series
Better In Vitro Cancer Models for Better Cancer Drugs
MILs Role in Biology and Cancer Therapy

ACKNOWLEDGMENTS

Hosted by: Gina Mullane
Narrated by: Chris Garcia
Special thanks to: Kim Noonan

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 Damage Repair and Huntington’s Disease
Factor-H South America
Dancing at the Vatican
Ignacio Muñoz-Sanjuan Speaks About Factor-H
Accelerating Therapies for Huntington’s Disease
Factor-H, Strengthening Communities Afflicted by HD in Latin America

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 LINK

OTHER 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 Garcia

Narrated by: Gina Mullane and Chris Garcia

Special thanks to:

Ian Waddell
Vad Lazari
Barbara 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 Garcia

Narrated by: Gina Mullane

Special thanks to:

Dr. David Fajgenbaum
Barbara Killian

The discovery of limulus amebocyte lysate (LAL) in the blood of horseshoe crabs has forever changed bacterial endotoxin testing. Today, researchers continue to look for any way they can protect these seafaring creatures while refining testing methods to decrease the need for animals. Join our panel for an in-depth look at how they’re working with legislators to preserve the horseshoe crab population and how LAL testing is the safest in vitro method to detect harmful bacteria.

Acknowledgements:

Hosted by: Gina Mullane

Narrated by: Chris Garcia

Special thanks to:

Dr. Norman Wainwright
Nicola Reid

Three-dimensional cell models are primed to play a vital role in the future of drug development. By recreating tissue that mimics human systems, scientists can monitor disease progression and evaluate the effects of drug candidates. Join Drs. Elizabeth Anderson and Ian Waddell as they explain the evolution of biologic modeling and how 3D cell models are being used to develop effective therapeutics to patients.

Acknowledgements:

Hosted by: Gina Mullane

Narrated by: Chris Garcia

Special thanks to:

Dr. Elizabeth Anderson
Dr. Ian Waddel

Within the past decade, CRISPR/Cas9 gene editing of mammalian cells has become common practice, but does it hold the key to unlocking future of therapeutic development? Join Dr. David Fischer and our panel for a detailed look at the advantages and applications of this Nobel Prize-winning technology.

Acknowledgements:

Hosted by: Gina Mullane

Narrated by: Chris Garcia

Special thanks to:

Dr. David Fischer

Get your four-part dose of vaccine…information, that is. In this brief recap, Vital Science hosts Gina Mullane and Chris Garcia chat about the content of our first several Season 2 episodes – a miniseries covering infectious disease and immunity, origins of vaccination, therapeutic development and regulation, and the scientific community’s recent accomplishments in the fight against COVID.

Download individual episodes:

See what you might have missed in the vaccine series or download and save individual episodes on our Season 2 tab.

Beyond the podcast:

Season 1 of Vital Science™ shared numerous compelling stories, like that of the Hermstad twins and their battle with ALS (Episode 7, An Uncommon Bond). Sadly, Jaci passed away shortly after her podcast aired, but the fight continues as clinicians, advocates, and drug developers continue to work in her honor. As Chris mentions in the recap, if you’re interested in the next chapter of her story, you won’t want to miss a free virtual event with our special guests Ben Stiller and Project ALS:

The Hermstad Legacy: Advances in Treatment for ALS

A Rare Disease Trailblazer Event

Thursday, February 4 | 11 a.m. – 4:00 p.m. ET

Register

Acknowledgements:

Hosted by: Gina Mullane and Chris Garcia

Recent developments in the race to develop a COVID vaccine show promise, but the question remains – when will it become available to the general public? Dr. Sarah Gould returns for the fourth and final installment of our vaccine miniseries to examine where we stand with the development of COVID-19 vaccines and some lingering challenges that may hamper their final approval.

Acknowledgements:

Hosted by: Chris Garcia

Narrated by: Gina Mullane

Special thanks to:

Dr. Sarah Gould

“Accelerating” a vaccine for COVID-19 has piqued the interest of many, but is it really possible to develop one quickly and safely? Join distinguished scientist Dr. Lauren Black as she dives into the typical mechanisms behind a vaccine and why it takes several years to deliver one safely to the general public.

Acknowledgements:

Hosted by: Chris Garcia

Narrated by: Gina Mullane

Special thanks to:

Dr. Lauren Black

When the immune system is compromised, people rely on vaccines to eliminate and inactivate the threat. However, the process with which this happens is anything but simple. Dr. Christina Satterwhite joins us to explain how the immune system works and how vaccines target harmful pathogens in the second installment of our series on vaccines.

Acknowledgements:

Hosted by: Chris Garcia

Narrated by: Gina Mullane

Special thanks to:

Dr. Christina Satterwhite

Since 1796, vaccines have helped humans combat smallpox, the flu, and other infectious diseases. The process of vaccine development has changed drastically since then, and is now front and center in the wake of COVID-19. In the first episode of our series on vaccines, join us as we talk with Dr. Sarah Gould about the history of vaccines and infectious disease.

Acknowledgements:

Hosted by: Chris Garcia

Narrated by: Gina Mullane

Special thanks to:

Dr. Sarah Gould

Cate and Laura Cheevers are sisters who were both born with cystic fibrosis. Together, they’ve been through multiple clinical trials for genotype-specific treatments and continue to defy the odds. Hear their unique tales of growing up with this rare genetic disorder and how it’s shaped their lives today.

Learn more about how the Cystic Fibrosis Foundation and Vertex Pharmaceuticals are working on treatments to aid those living with CF, and support Great Strides on their quest to find a cure for CF.

Acknowledgements:

Hosted by: Chris Garcia

Produced and Edited by: Meaghan Root and Ryan Stevenson

Sound and Music by: Ryan Stevenson

Special thanks to:

Kim, Cate, and Laura Cheevers

Dr. Guy Leclerc plays the role of both a researcher and a clinician, changing lives with his work on medical devices. Learn how his work directly improved the quality of life for a very special patient.

Learn more about Isabelle’s story, and support what drives Dr. Leclerc by donating to the CHUM foundation.

ACKNOWLEDGMENTS
Hosted by: Gina Mullane
Produced and Edited by: Meaghan Root and Ryan Stevenson
Sound and Music by: David Coscia and Ryan Stevenson
Special thanks to: Dr. Guy Leclerc, Isabelle

Jake Glanville, founding partner and CEO of Distributed Bio, has been featured in the Netflix series "Pandemic." Now, he and his team are on the front lines of the fight against COVID-19, working tirelessly to find a cure.

Learn how his roots in Guatemala have shaped his beliefs on healthcare, how a family business has developed his skills as a leader, and how it all came together in his leadership of a talented, accomplished team of scientists.

Read more about antibody discovery technologies and advancements.

ACKNOWLEDGMENTS

Hosted by: Gina Mullane
Produced and Edited by: Meaghan Root, Ryan Stevenson, and Pam Williams
Sound and Music by: David Coscia and Ryan Stevenson

Special thanks to: Jacob Glanville, PhD

Twins share many unique bonds, and for Jaci and Alex, fighting the same rare disease is one of them. Hear this emotional tale through the perspective of their mom, Lori, and their uncle, Mike, about how they have battled their illness, knowing the risks, and helped to develop a new treatment for this rare form of ALS.

To learn more about Jaci’s story, please visit Cowgirl Up for Jaci: Roping in a CURE for ALS. For more information about ALS, please visit Project ALS.

ACKNOWLEDGMENTS

Hosted by: Chris Garcia
Produced by: Meaghan Root, Ryan Stevenson, and Laura Robinson
Sound and Music by: Ryan Stevenson

Special thanks to: Lori Hermstad, and Mike Hadden
In memoriam: Jaci Hermstad and Alex Hermstad

College can take you many places. In the case of two neuroscience students, they didn’t know where their journey would take them. Their perseverance and fighting spirit built a company that aims to help those battling neurodegenerative diseases.

See what Amylyx is working on.

There are certain moments when life takes on a whole new meaning. For Allyson Berent, her daughter’s diagnosis with a rare genetic disorder gave her a purpose – to embark on the quest for a cure. Hear her remarkable journey from a veterinarian to the COO of GeneTx Biotherapeutics, and how partnering with Ultragenyx pharmaceutical fuels her determination to cure Angelman Syndrome.

Learn more about FAST (Foundation for Angelman Syndrome Therapeutics).

What we experience can leave a lasting impact on our lives. In Carina’s case, her father’s diagnosis changed her career forever. Hear the emotional story of her ongoing fight to honor his memory and help others battling this rare neurodegenerative disease.

Learn more about the Multiple System Atrophy Coalition

Donnie Patterson is not just a pharmaceutical microbiologist. He’s also a victim of toxigenic mold, a growing problem in South Carolina schools. Hear how his story exposed an issue that led to his ongoing fight for tougher mold testing regulations.

Visit the Global Indoor Health Network website for more information on toxic mold.

Mila’s groundbreaking treatment, the first created and approved for a single patient, has given hope to thousands of rare-disease patients around the world. Hear Julia Vitarello, Mila’s Mom, and Dr. Timothy Yu discuss their plans to help other children with rare disease.

Watch the documentary and learn more about Mila’s story at: www.criver.com/everystep

In partnership with Mila’s Miracle Foundation, we are on a mission to bring this new treatment path to children fighting rare diseases. An anonymous donor will match up to $150,000 in donations to help with this fight, and Charles River has already donated $50,000 to get the campaign started.

Join our giving campaign

ACKNOWLEDGMENTS

Hosted by: Gina Mullane and Chris Garcia
Produced by: Meaghan Root and Ryan Stevenson
Sound and Music by: Ryan Stevenson

Special thanks to: Julia Vitarello, Dr. Timothy Yu, and Boston Children’s Hospital.

Mila is a young girl fighting Batten Disease, a rare, fatal disease with no cure. Her Mom, Julia Vitarello sits down with Gina to tell the story of Mila’s diagnosis and her incredible journey since; from diagnosis to Mila’s experimental genetic treatment, created just for her.

Watch the documentary and learn more about Mila’s story at www.criver.com/everystep.

Hosted by: Gina Mullane and Chris Garcia
Produced by: Meaghan Root, Ryan Stevenson
Sound and Music by: Ryan Stevenson
Special thanks to: Julia Vitarello, Dr. Timothy Yu, and Boston Children’s Hospital.

Meet our hosts Gina Mullane and Chris Garcia as they introduce Vital Science™, a brand new podcast that shares the personal stories of those impacted by life science. Subscribe so you don't miss us, starting October 2019.

ACKNOWLEDGMENTS

Hosted by: Gina Mullane and Chris Garcia
Produced by: Meaghan Root and Ryan Stevenson
Sound and Music by: Ryan Stevenson