Vital Science Charles River

At Mission Therapeutics, Sarah Almond serves as their Head of Pharmacology, but she wears many hats to help develop potential treatments for neurodegenerative conditions.    
  
Their main area of focus centers around research into deubiquitinating enzymes (DUBs), which may impact neurodegeneration. By studying DUBs, Sarah and her team have been able to generate highly targeted and potent molecules that contribute to developing safe and effective therapeutics. In fact, one of these compounds (MTX325) is currently enrolled in a clinical trial to test its safety and pharmacokinetics ahead of effectiveness in treating Parkinson’s disease, a neurodegenerative condition impacting the central nervous system. With the first patient dosing scheduled for later this year, they are hopeful it could be a gamechanger for treating these debilitating conditions.    
  
Join Sarah as we discuss Mission Therapeutics, the science behind DUBs, how collaboration with a CRO advanced their research, and what her thoughts are on the future of drug discovery and development for neurodegenerative conditions, among others.
Show Notes
Mission Therapeutics Poster: Development and validation of a high content-based assay to measure Tom20 loss in dopaminergic human neurons differentiated in vitro Parkinson's Disease Studies | Charles River Neuroscience | Charles River Knockout or Inhibition of USP30 protects Dopaminergic Neurons in a Parkinson's Disease Mouse Model Mission Therapeutics granted MHRA Clinical Trial Authorisation (CTA) for MTX325 for the treatment of Parkinson’s Disease Mission Therapeutics announces US FDA approval to initiate Phase II clinical trial of its lead asset MTX652 in Acute Kidney Injury 

When he founded Cure Rare Disease in 2018, Rich Horgan was primarily focused on finding a treatment for his younger brother Terry, who had an ultra-rare form of Duchenne Muscular Dystrophy (DMD).  
 
Suffering from a disease that has no cure, Terry served as a “selfless, brave, and courageous” figure that Rich continues to draw inspiration from when seeking new, innovative solutions to treat this progressive, neurodegenerative condition.  
 
Join us for this interview as Rich discusses the origins of Cure Rare Disease, Terry’s ongoing legacy, the collaborative effort it takes to advance the drug development journey, the important role of the patient voice when developing treatments, and the future of rare disease research. 
Show Notes 
What We Owe Terry Horgan: Reflections from Providers, Family, and Scientists Letter From Terry's Parents The Disruptors - Meet Rich In Vitro Assays for Duchenne Muscular Dystrophy 2022 World Congress Video Duchenne Muscular Dystrophy Studies Rare Disease Research for Drug Development Scientific Collaboration Leads to Faster Drug Development Cure Rare Disease   

For the 850 million people around the world suffering from chronic kidney disease, the grind of dialysis treatment can wear them down in more ways than one.   
 
Dr. Andy Herbert, co-founder of Invizius, is out to change that. This five-year-old company started from humble beginnings and is currently developing H-Guard® Priming Solution, which increases the effectiveness of dialysis while negating, or eliminating, its potentially dangerous side effects. It will be used in an upcoming first-in-human clinical trial and has the potential to improve patients’ quality of life – both physically and mentally.  
 
Join Dr. Herbert as we discuss the origins of Invizius, how H-Guard works, why dialysis can take a toll on mental health, and how working with a strategic partner has helped Invizius progress this treatment.
Show Notes  
 Invizius 
Charles River's Nonclinical Support Helps Invizius Secure MHRA Approval for First-in-Human Trial of H-Guard® Hemodialysis Solution 
Renal Disorders | Charles River 
Preclinical CRO Services for Safety Assessment 
Inflammation | Charles River

Over the past 12 months, we have shared some remarkable stories from the scientific community, but three of the most impactful have come from “Disruptors” in their respective fields. Relive these stories with our hosts on the year-end edition of Vital Science.
Show Notes  
The Disruptors: Conversations in Science Meet Valerie Meet Wise Charles River | Gene Therapy for Neurological Disorders In Vitro and In Vivo Model of Amyotrophic Lateral Sclerosis  https://www.criver.com/eureka/neuroinflammation-in-spinal-cord-injury-a-necessary-evil W.M. Keck Center for Collaborative Neuroscience   

When Maria Picone’s daughter was diagnosed with Prader-Willi Syndrome (PWS), she turned to the internet for answers.  
 
What she found was an online community of caregivers sharing their experiences with PWS, giving her comfort and direction with what to do about her daughter’s rare disease. It also spurred her and her husband to create TREND Community, an online platform for caregivers of rare diseases to not only share their experiences, but also make positive contributions to scientific literature.  
 
Join us as we discuss the philosophy of TREND Community, how it transforms general discussions into real-world clinical research, how collaboration drives their progress, what’s next for TREND, and what you can do to help advance their mission.   
 Show Notes
 
Trend Community - Impact Through Insights
Trend Community Report on Identifying Mental Health Challenges Experienced by Caregivers in Rare Disease Communities: A Social Media Analysis
Charles River | Rare Disease
The Disruptors: Innovation in Drug Discovery

Imagine a world where your child has a rare disease that’s so rare, it doesn’t have a name and only a handful of people around the world have a similar condition. It can leave you feeling helpless and alone knowing the odds are stacked against you. 
 
This is the situation Bo Bigelow experienced with his daughter, Tess. She suffers from a USP7, a neurodevelopmental disorder that impacts her motor function, speech, and bone development. Tess’ condition left Bo looking for help… and he found a lot of other patients and families with other ultra-rare diseases that wanted to share their stories too. Today, Bo’s initiatives have given hope to other people and help them find strength in knowing they’re not alone. 
 
Join us as we discuss the ways Bo documented his daughter’s journey, learn more about USP7-related diseases and their impact, how Bo created the Disorder Channel as a way for other rare disease patients to tell their stories, what progress is being made to learn more about Tess’ condition through the Foundation for USP7-Related Diseases, and how you can help advance their research. 
Show Notes
 
Rare Disease | Charles River
Foundation for USP-7 Related Diseases
Stronger Every Day Blog
Stronger Every Day Podcast
The Disorder Channel