Spastic Paraplegia, Hereditary

Company will perform plasmid DNA production in support of early phase trials for SPG56 Hereditary Spastic Paraplegia WILMINGTON, Mass.--(BUSINESS WIRE)-- Charles River Laboratories International, Inc. (NYSE: CRL) and Genetic Cures for Kids Inc (GC4K), an Australian non-profit foundation focused on research programs and clinical trials to find cures for rare diseases, today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration. Founded in 2021 by Golden and Chris Whitrod, parents of now 4 year old Tallulah Moon, GC4K’s first mission, known as Our Moon’s Mission, is to develop a gene therapy for the treatment of Hereditary Spastic Paraplegia Type 56 (SPG56). SPG56 is a debilitating neurodegenerative disease that robs children of the ability to walk, talk, and move independently. What is SPG56? SPG56 manifests as a progressive neurological disease characterised by varying degrees of spasticity and muscle weakness. This degenerative disease typically begins with motor and cognitive regression in childhood and continuously worsens through life. Additional clinical manifestations include intellectual disability, dystonia, cerebellar ataxia, subclinical peripheral neuropathy, seizures, and visual impairment. Tragically, there is currently no treatment for this ultra-rare genetic disease. In addition, limited access to genetic testing means diagnoses are hard to achieve and as such, fewer than one in 1 million people have ever been diagnosed with SPG56 globally. GC4K is leading a gene therapy program that aim not to only cure SPG56, but to create a replicable framework that paves the way to develop treatments for some of the 7,000 other genetic diseases in the world. Plasmid DNA Manufacturing Services GC4K will leverage Charles River’s established plasmid platform, eXpDNA™, and premier expertise in plasmid DNA production, including High Quality (HQ) plasmid which combines key features of good manufacturing practice (GMP) manufacture with a rapid turnaround to accelerate time to clinic. As a critical starting material in the development of cell and gene therapies, in 2022 Charles River opened a state-of-the-art HQ Plasmid Manufacturing Center of Excellence dedicated to addressing global supply shortages and support the growing needs of customers. In recent years, Charles River has significantly broadened its cell and gene therapy portfolio to simplify complex supply chains and meet growing demand for plasmid DNA, viral vector, and cell therapy services. Combined with the Company’s legacy testing capabilities, Charles River offers a comprehensive “concept-to-cure” advanced therapies solution. To learn more about plasmid DNA production, watch Advancements in Manufacturing for Rapid Delivery of pDNA Starting Materials, presented by Andrew Frazer, PhD, Associate Director, Scientific Solutions, Gene Therapy CDMO Services, on-demand: Approved Quotes “The opportunity to work with GC4K is exactly why we do what we do, playing a role in delivering potentially curative treatments to patients suffering with an ultra-rare disease. We are excited to support the team as they work through the next stage of their program development.” - Kerstin Dolph, Corporate Senior Vice President, Biologics Solutions, Charles River “What was once a tragic diagnosis, has the potential to become a triumph, as we draw nearer to finding a cure for SPG56. With the advancement of this groundbreaking treatment, our partnership with Charles River is bringing us closer to aiding children like Tallulah, who, until now, have faced the daily hardships of SPG56 without any glimmer of hope. We're truly excited to have the backing of this skilled team as we gear up to produce this historic treatment. By making this announcement on Giving Tuesday, we are giving thanks to Charles River. This symbolic occasion not only commends the generosity of CRL in supporting GC4K’s quest to cure SPG56, but also to recognizes the broader goal that our partnership is striving for - a brighter future for all children living with rare genetic diseases." - Golden Whitrod, Tallulah Moon’s mother and Co-Founder and President, Genetic Cures for Kids, Inc. About Charles River Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit . Genetic Cures for Kids, Inc., and Our Moon’s Mission GC4K is a family-driven research foundation at the forefront of genetic research, with a primary objective to not only develop a cure for SPG56 but also to establish a reproducible framework that can catalyze the development of treatments for some of the 7,000 known genetic diseases worldwide. The foundation's campaign to cure SPG56 is "Our Moon's Mission", named in honor of the founders’ daughter Tallulah Moon, who lives with SPG56. By leveraging crowdfunding to assemble an expert international research team, which has since gained recognition and funding from the Australian Government and is guided by an Independent Scientific Advisory Board, GC4K aims to expedite the development of effective SPG56 therapies for clinical use. Additionally, the foundation is dedicated to advocating for 'rare families' and increasing awareness of rare diseases. To learn more about GC4K's commitment to advancing genetic research and to support the foundation's mission to cure SPG56, please visit

THURSDAY, July 27, 2023 -- Many parents in the United States aren't installing child car seats correctly, a new study finds. Errors in car seat installation are common, even for seats that have a 5-star rating for features like ease of use, researchers found. The study found that fewer errors were detected when parents installed seats that had higher ratings, but researchers recommend that parents seek out safety technicians to learn the proper techniques for seat installation. Child restraint systems cut the risk of crash injuries by 50% to 85%, but only if properly used, the researchers noted. "New parents often receive training on car seat installation before the baby is born," said researcher Dr. Michelle Macy , an emergency medicine physician at Ann & Robert H. Lurie Children’s Hospital of Chicago. "However, it would be beneficial for them to take advantage of the available resources after the child’s birth as well, especially during the transition from infant carrier to a rear-facing car seat, and then again when switching the seat to face forward," she said in a hospital news release. For the study, Macy and her colleagues analyzed data from Safe Kids Illinois seat check records from 2015 through 2019. The most common errors were for seats installed with seat belts (70%) and the least common for recline angle (37%). One of the more common errors that were found around 50% of the time, even with 5-star rated car seats, involved the top tether on forward-facing car seats. The top tether is the strap on top of the seat that needs to be hooked to an anchor point on the vehicle. Often parents do not attach the strap or hook it in the wrong location. "Overall, our study results show that parents can rely on the car seat rating system when choosing an appropriate car seat for their child," Macy said. "They just need to be aware that installation and use errors can still occur even with the top-rated car seats. We encourage parents to get help from a certified technician to ensure their child’s safety on the road." The report was published July 21 in the journal Traffic Injury Prevention . The National Highway Traffic Safety Administration website provides a resource directory by zip code to help parents connect with a nearby certified child passenger safety technician. Sources Ann and Robert H. Lurie Children's Hospital of Chicago, news release, July 21, 2023 Posted July 2023

Errors in installation of child car seats are common, even with seats that have a five-star rating for ease of use, according to a new study. The study found that although the rating system was a suitable indicator of ease of use, with fewer errors detected when parents installed seats that had higher ratings, more efforts are needed to ensure optimal safety for young passengers. Errors in installation of child car seats are common, even with seats that have a five-star rating for ease of use, according to a study published in the journal Traffic Injury Prevention. The study found that although the rating system was a suitable indicator of ease of use, with fewer errors detected when parents installed seats that had higher ratings, more efforts are needed to ensure optimal safety for young passengers. Child restraint systems (CRSs) reduce risk of crash-related injury by 50%-85%, however use errors undermine their benefits. The National Highway Traffic Safety Administration (NHTSA) created the Ease of Use (EOU) rating system to help guide consumers and incentivize manufacturers to improve their products. The EOU rating system assigns one to five stars to four CRS features and overall. Child passenger safety technicians working in the community and with car seat manufacturers are available to answer questions about installing and using car seats. The NHTSA website provides a resource directory by zip code to help parents connect with a local certified child passenger safety technician who can check if the seat is installed correctly or provide help on installing and using an appropriate car seat. "New parents often receive training on car seat installation before the baby is born. However, it would be beneficial for them to take advantage of the available resources after the child's birth as well, especially during the transition from infant carrier to rear-facing car seat, and then again when switching to the seat to face forward," said lead author Michelle Macy, MD, MS, Emergency Medicine physician at Ann & Robert H. Lurie Children's Hospital of Chicago and Associate Professor of Pediatrics at Northwestern University Feinberg School of Medicine. Dr. Macy and colleagues analyzed data from Safe Kids Illinois seat check records from 2015 through 2019 and EOU ratings from 2008 to 2020. Errors were most common for seats installed with seat belts (70%) and least common for recline angle (37%). One of the more common errors that was found around 50% of the time, even with 5-star rated car seats, involved the top tether on forward-facing car seats. The top tether is a strap on the top of the seat that needs to be hooked to an anchor point on the vehicle. Often parents either do not attach the strap or hook it to the wrong location. "Overall, our study results show that parents can rely on the car seat rating system when choosing an appropriate car seat for their child," said Dr. Macy. "They just need to be aware that installation and use errors can still occur even with the top-rated car seats. We encourage parents to get help from a certified technician to ensure their child's safety on the road." Dr. Macy holds the Mary Ann & J. Milburn Smith Research Professorship for the Director of Child Health Research. She is the Director of Mary Ann & J. Milburn Smith Child Health Outcomes, Research and Evaluation Center; and Scientific Director of Community, Population Health, and Outcomes at Stanley Manne Children's Research Institute. Research at Ann & Robert H. Lurie Children's Hospital of Chicago is conducted through Stanley Manne Children's Research Institute. The Manne Research Institute is focused on improving child health, transforming pediatric medicine and ensuring healthier futures through the relentless pursuit of knowledge. Lurie Children's is a nonprofit organization committed to providing access to exceptional care for every child. It is ranked as one of the nation's top children's hospitals by U.S. News & World Report. Lurie Children's is the pediatric training ground for Northwestern University Feinberg School of Medicine. Emergency medicine-focused research at Lurie Children's is conducted through the Grainger Research Program in Pediatric Emergency Medicine.