Vital Science Charles River

Imagine giving birth to a baby girl and noticing pieces of skin missing from her body almost immediately.  
For Sharmila Nikapota, this was the case with her firstborn, Sohana. Genetic testing revealed that she was one of the over 500,000 people globally suffering from Epidermolysis Bullosa (EB), a “constantly painful and debilitating” skin condition where minimal contact can lead to blisters, wounds, tissue damage, eating difficulties, and eye injuries, among other symptoms. After seeking answers for her daughter’s prognosis and not getting any, Sharmila started Cure EB to educate people and eventually discover ways to manage this painful condition. With the UK approving a first-of-its-kind topical treatment for EB, she hopes it’s the first of many that can give patients like her daughter a better quality of life.
Join Sharmila as she discusses how Sohana’s experience with EB has shaped her personality, the origin and mission of Cure EB, the challenges of managing Sohana’s condition, and what you can do to further Cure EB’s ultimate goals.
Show Notes 
Cure EB - Accelerating Research to End Painful SkinBirch Bark Extract: A Review in Epidermis BullosaCharles River | Cell and Gene TherapyCharles River | Rare DiseaseEureka Blog | Living RareKrystal Biotech Touts Topical Gene Therapy Uptake

Stimulant use in the United States has exploded into a nationwide crisis, as deaths and emergency room visits resulting from methamphetamine and cocaine use are rising at an alarming rate.
The overall substance abuse disorder crisis is further exacerbated by the high level of fentanyl use, which has long been referred to as an opioid epidemic, and the increasing use of more than substance at once. One fundamental limitation on the ability to respond to the crisis is that, to date, there is no FDA-approved therapeutic to treat overdose from stimulants like methamphetamine and cocaine, or polydrug. Users who want to get clean and rid themselves of methamphetamine, cocaine, and/or fentanyl often find it difficult, essentially “chaining” themselves into a cycle of use that’s mostly impossible to break.
First responders and emergency department physicians lack sufficient tools to treat overdose. But what if there was a treatment that could eliminate toxins and restore normal bodily function within minutes? That’s the aim of Clear Scientific, a small biotech in Cambridge, MA that’s currently testing a treatment (CS-1103) using small molecules to “eat” toxins and deactivate their harmful effects almost instantly. With Phase I of a first-in-human (FIH) clinical trial ongoing, this revolutionary therapeutic is offering hope to those suffering from drug misuse and addiction a “chance to get better.”
Join us as Mitch Zakin, Co-founder and Executive Vice President of Innovation, and Winston Henderson, Co-founder and General Counsel, discuss their therapeutic approach, offer an update on their progress with CS-1103, how collaboration has enhanced their development, and how they hope it will eventually impact this epidemic.
Show Notes  

Clear Scientific 
World Congress: Drug Modality Game Changers 
Clear Scientific to Start Phase 1 Trial for Stimulant Antidote 
Charles River | IND-Enabling Studies 
Charles River | Small Molecule 

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