Open Science: The Parallel Universe

The effects of Angelman Syndrome (AS) on young children are debilitating. From an early age, they face an uphill battle with this neurogenetic disease’s unrelenting symptoms, including seizures, cognitive impairments, and loss of motor function. It can also be tough on caretakers – just ask Dr. Allyson Berent and Jennifer Panagoulias. They both have personal connections to this disease, which in Dr. Berent’s case led to her creating the Foundation for Angelman Syndrome Therapeutics (FAST) in 2008. Today, Panagoulias, whose niece has AS, serves as their head of Regulatory and Policy, tasked with the responsibility to find a path that will develop treatments to improve the quality of life for both patients and caregivers.  Join Jennifer as we discuss the origins of FAST, the science behind Angelman Syndrome, the crucial role outside partnerships have played in research, and what’s in the pipeline for potential therapeutics.Show Notes  Foundation for Angelman Syndrome Therapeutics  ASOs Rescue Brain Rhythms, Sleep Patterns in Angelman Mouse Model  A Big Year for Angelman Syndrome  Antisense Therapies and Angelman's Syndrome  Unsilencing Quincy  The Quest to Cure Quincy  Development of Potential Outcome Measures for AS Clinical Trials – Angelman Syndrome Foundation   

When patients face the diagnosis of a rare disease, they face a costly, uphill battle to develop a treatment for their condition… that is, if they ever do.   Dr. Stanley Crooke is aiming to change that. As the CEO of n-Lorem Foundation, his mission is to provide personalized treatments to ultra-rare disease patients for free as long as they live, no questions asked. Thanks to his humility, coupled with the generosity of other individuals and companies, ultra-rare disease patients have a renewed hope to get the treatments others may not be able to provide.   Join Dr. Crooke as he reflects on his humble beginnings at Ionis, the approach n-Lorem takes to developing drugs for ultra-rare diseases, and what the future holds for research in this area.Show Notes  n-lorem Foundation  Charles River - Rare Disease  Vital Science S4, E01 Project ALS: Fueled by Love  2021 Rare Disease Trailblazers  BioSpace: n-Lorem Foundation Preps First Doses to Treat Ultra-Rate Disease Patients for Free  Ionis Pharmaceuticals  Nature Medicine: Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis  

Imagine being diagnosed with an incurable rare disease before ever being born. This is the reality that Evie faced when she was diagnosed with hypophosphatasia, a condition impacting bone growth, breathing, and mobility, and often gives patients just 18 months to live.    Luckily, her story doesn’t end there. At three months old, she enrolled in a clinical trial for a first-of-its-kind medication that her mom Lindsey claims “probably saved her life”. Today, Evie is 13 years old and continues to defy the odds, giving hope to other young children suffering from a rare disease that they too can find the strength to overcome anything life throws at them.   Join Evie and Lindsey for this emotional look at their journey, how the power of collaboration has helped them along the way, and Evie’s goals for the future.Show Notes  Every Patient: Evie  Evie's Story  Rare Disease Impact  Charles River Partners with Soft Bones: The US Hypophosphatasia Foundation  Probing the Genes of Rare Disease  No Time To Waste: Collaborative Efforts in Rare Disease Drug Discovery  Charles River Discovery Services 

Could it be possible to regenerate neurons in the human body?  It’s a question that has driven Dr. Wise Young for over 40 years. He’s dedicated his career to researching spinal cord injuries and how to develop a treatment for them. Along the way, he’s discovered neurogenesis, a way to regenerate neurons in not just the spinal cord, but also the brain. Thanks to his work, what everybody told him was once impossible, is now close to becoming a reality.   Join us as Dr. Young discusses his founding of, and work at, the W.M. Keck Center for Collaborative Neuroscience, how the components of stem cells and umbilical cord blood can help regenerate neurons, and what his vision is for the future of research in this growing field.Show Notes  The Disruptors - Meet Wise  https://www.criver.com/therapeutic-area/neuroscience  https://www.criver.com/products-services/research-models-services  https://www.criver.com/eureka/neuroinflammation-in-spinal-cord-injury-a-necessary-evil  https://www.criver.com/eureka/making-efficacy-models-count  https://www.criver.com/molecule-type/cell-and-gene-therapy-services  https://www.criver.com/products-services/find-model/rnu-nude-rat?region=3611  https://www.criver.com/products-services/safety-assessment  https://www.criver.com/industry/academic

Recent advancements in research has led to the development of effective therapeutics to combat neurodegenerative diseases, giving patients a renewed sense of hope there will ultimately be a more effective treatment.CEO Lisa Deschamps and her team at AviadoBio are among those conducting research in the space, but they’re doing things a little differently. They’ve been given a unique drug designation by the FDA to develop a treatment for frontotemporal dementia (FTD), a crippling neurodegenerative disorder that affects neuron production in people under 65. It may be a “bold and courageous” undertaking, but the benefits could affect future generations to come.Join us as we take a look at the motivation behind the work AviadoBio is doing, what makes their therapy unique, and Lisa’s vision for their future.Show Notes  AviadoBio  S4, E01: Project ALS: Fueled by Love  S3, E01: A Synergistic Approach to Treating Huntington's Disease  Charles River | Neuroscience  Charles River | Gene Therapy for Neurological Disorders  Charles River| GEne Therapy Services  

As we close in on the end of 2022, we’ve had the privilege to interview some amazing people who are doing great work with drug discovery.   Join Gina Mullane and Todd Poley as we reflect on some of the memorable moments and guests from this season, including an update on Amylyx Pharmaceuticals’ new ALS treatment, Chris Claussen’s discussion on the positive impacts of psychedelic drugs, and the work of Project ALS to develop a treatment for this crippling disease. On behalf of our entire team, thank you for listening this year. We hope you’ll join us for an exciting lineup of remarkable stories set to premiere in 2023.Show Notes: Amylyx Pharmaceuticals Announces FDA Approval of RELYVRIO™ for the Treatment of ALS  Alzheimer’s Disease Studies   In Vitro and In Vivo Models of Amyotrophic Lateral Sclerosis  Are Psychedelics the Next Approach to Treating Mental Health Disorders?  FIRST PERSON LAUNCHES INNOVATIVE PRECISION-TARGETED COGNITIVE SUPPLEMENTS TO ACTIVATE THE FULL POTENTIAL OF HUMAN COGNITION  The Hermstad Legacy: Advances in Treatments for ALS   Project ALS  

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