CORRECTING and REPLACING ASC Therapeutics, UMass Chan Medical School, and the Clinic for Special Children Announce Podium Presentation of Safety and…
MILPITAS, Calif.--(BUSINESS WIRE)--Please replace the release dated May 16, 2022 with the following corrected version due to multiple revisions.
The updated release reads:
ASC THERAPEUTICS, UMASS CHAN MEDICAL SCHOOL, AND THE CLINIC FOR SPECIAL CHILDREN ANNOUNCE PODIUM PRESENTATION OF SAFETY AND EFFICACY IN MURINE AND BOVINE MODELS FOR NOVEL GENE THERAPY IN MAPLE SYRUP URINE DISEASE AT THE 25TH ASGCT MEETING
ASC Therapeutics in partnership with the UMass Chan Medical School and the Clinic for Special Children (CSC), presented safety and efficacy results of a dual-function gene replacement vector therapy in murine and bovine models of classic Maple Syrup Urine Disease (MSUD) as a podium presentation at the 25th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) held May 16-19, 2022 in Washington D.C.
The research group at UMass Chan, led by Guangping Gao, PhD, director of the Horae Gene Therapy Center at UMass Chan, and Dan Wang, PhD, assistant professor of RNA therapeutics at UMass Chan, developed murine and bovine models for MSUD with collaborating clinical expert, Dr. Kevin Strauss, MD, from the Clinic for Special Children.
Three animal models were generated to test safety and efficacy: two murine models representing two common genetic forms of MSUD and a newborn calf naturally homozygous for a mutation that causes MSUD. All animals exhibited severe biochemical abnormalities hours after birth and die within 10 days if left untreated.
The partnership designed a dual-function AAV9 gene replacement vector that was administered via intravenous (IV) injection. Surviving mice grew and behaved similar to normal littermates and had normal or nearly normal biochemical markers with unrestricted diet for 16 weeks.
As compared to MSUD patients, MSUD calves exhibit a similar phenotype and are closely matched for size and metabolic rate. Without treatment they exhibit cerebral edema by day of life 3 and die soon after. In October 2021, a MSUD calf was born and developed biochemical signs of MSUD shortly after birth. The calf was administered the AAV9 gene therapy vector at 40 hours of life. Following AAV therapy, biomarker measurements indicated a significant restoration of the missing enzymatic activity. At 70 days after treatment, the calf was transitioned to a normal unrestricted diet and continues to thrive 100 days post-infusion with stable biochemical markers.
These data provide early demonstration of the safety and efficacy of the MSUD AAV9 gene therapy replacement vector as a one-time treatment for the most common and severe forms of MSUD.
The MSUD gene therapy development leverages the combined AAV gene therapy expertise at UMass Chan Medical School, such as AAV vector design, rodent and large animal modeling, large-scale vector production, and in vivo pre-clinical testing, Drs. Gao and Wang commented.
Dr. Kevin Strauss, MD, Medical Director at the Clinic for Special Children in Pennsylvania, a collaborating clinical expert, added, An innovative collaboration between the Clinic for Special Children and UMass Chans Horae Gene Therapy Center has allowed us to thoughtfully streamline the process of developing AAV gene replacement vectors. Within just three years of project inception, we have safely corrected an otherwise fatal MSUD phenotype in both mice and a newborn calf using a novel dual-function BCKDHA-BCKDHB vector, which has the potential to address 70-80% of reported MSUD cases in humans. The newborn calf with MSUD may represent the largest non-human experimental animal ever treated with AAV-mediated gene replacement. The calf provides unique insights that can directly inform the design of a clinical trial, which we hope to pursue through an alliance with ASC Therapeutics."
Dr Ruhong Jiang, CEO at ASC Therapeutics, said, The significant progress achieved through our research collaboration with Professors Gao, Wang and Strauss underlines the intrinsic value of bringing together teams from academia and industry that are highly specialized in complex gene therapies.
About Maple Syrup Urine Disease
Maple syrup urine disease (MSUD) is a rare genetic disorder affecting degradation of the branched-chain amino acids (BCAA) leucine, isoleucine, and valine and their ketoacid derivatives. MSUD is caused by biallelic mutations in one of three genes that encode subunits of the branched-chain ketoacid dehydrogenase complex (BCKDC), namely BCKDHA, BCKDHB, and DBT. Dietary BCAA restriction is the mainstay of treatment but has insufficient efficacy, and affords no protection against episodic and life-threatening encephalopathic crises. Severe (classic) MSUD is fatal without treatment. MSUD affects approximately 1 per 185,000 births worldwide.
About ASC Therapeutics
ASC Therapeutics is a biopharmaceutical company pioneering the development of gene replacement therapies, in-vivo gene editing and allogeneic cell therapies for hematological, metabolic, and other rare diseases. Led by a management team of industry veterans with significant global experience in gene and cell therapy, ASC Therapeutics is developing multiple therapeutic programs based on four technology platforms: 1) In-vivo gene therapy of inherited blood clotting disorders, initially focusing on ASC618, a second generation gene replacement treatment for hemophilia A; 2) In-vivo gene therapy in metabolic disorders, initially focusing on Maple Syrup Urine Disease; 3) In-vivo gene editing, initially focusing on ASC518 for hemophilia A; and 4) Allogeneic cell therapy, initially focusing on a Decidua Stromal Cell-based therapy for steroid-refractory acute Graft-versus-Host Disease. To learn more please visit https://www.asctherapeutics.com/.
About the Clinic for Special Children
The Clinic for Special Children (CSC) is a non-profit organization located in Strasburg, PA, which provides primary care and advanced laboratory services to those who live with genetic or other complex medical disorders. Founded in 1989, the organization provides services to over 1,200 individuals and is recognized as a world-leader in translational and precision medicine. The organization is primarily supported through community fundraising events and donations. For more information, please visit http://www.ClinicforSpecialChildren.org
About UMass Chan Medical School
The UMass Chan Medical School, one of five campuses of the University of Massachusetts system, comprises the T.H. Chan School of Medicine, the Morningside Graduate School of Biomedical Sciences, the Tan Chingfen Graduate School of Nursing; a thriving research enterprise and an innovative public service initiative, Commonwealth Medicine. UMass Chans mission is to advance the health and wellness of our diverse communities throughout Massachusetts and across the world by leading and innovating in education, research, health care delivery and public service. In doing so, it has built a reputation as a world-class research institution and as a leader in primary care education, perennially ranked in the top 10 percent of medical schools for primary care by U.S. News and World Report. UMass Chan attracts more than $400 million annually in research funding, placing it among the top 50 medical schools in the nation. In 2021, the Medical School received a $175 million donation from The Morningside Foundation and was renamed the UMass Chan Medical School.
- How to Gain Weight Safely - Health Essentials from Cleveland Clinic - June 27th, 2022
- Following a Mediterranean Diet Could Reduce Alzheimer's and Diabetes by up to 50 Percent - The Epoch Times - June 27th, 2022
- Democracy and the Tribal Blame Machine - Justia Verdict - June 27th, 2022
- Nordic walking helps improve heart function, study finds - Medical News Today - June 27th, 2022
- 5 Exercises That The Elderly Must Do At Home To Maintain Good Health | TheHealthSite.com - TheHealthSite - June 27th, 2022
- Stomach Pain and Type 1 Diabetes: What You Should Know - Healthline - June 27th, 2022
- Drivers advised to give turtles a 'brake' - BradfordToday - June 27th, 2022
- Why heart diseases are on the rise among young Indians - The Week - June 27th, 2022
- Here are 16 alders who voted to allow deadly speeds, and crash cases in their wards. - Streetsblog Chicago - June 27th, 2022
- Beyond gluten-free: The promising celiac treatments on the horizon - The Daily Briefing - June 8th, 2022
- Eating all meals before 3pm and nothing in the evening helps weight loss, study says - The Mirror - June 8th, 2022
- Don't throw away the peel! Skins of fruits and vegetables that are safe to eat - Times Now - June 8th, 2022
- Good bacteria in the treatment of Crohn's disease - Medical News Today - June 8th, 2022
- What To Do if Youre Worried About Your Childs Safety at School or Youve Experienced a Crisis - Health Essentials from Cleveland Clinic - June 8th, 2022
- Five 'dental health tips' that could actually ruin your teeth, says top dentist - The Mirror - June 8th, 2022
- Studies Uncover Stark Impact Social Determinants of Health Have on Youth and Adult Populations with Diabetes - StreetInsider.com - June 8th, 2022
- Game-changing drug can help tubby Brits shed up to 52lbs, experts say... - The Sun - June 8th, 2022
- Intermittent fasting for women: Is it safe? - Livescience.com - May 21st, 2022
- Why it's OK to give your baby spicy food - and how to do it safely - Metro.co.uk - May 21st, 2022
- Active Fit: Building strong bones and muscles - The Newark Advocate - May 21st, 2022
- What to Know About Fentanyl and Why Its So Dangerous - The New York Times - May 21st, 2022
- How IBS is treated: diet and treatment of irritable bowel syndrome - Emergency Live International - May 21st, 2022
- Cast Iron Cooking: Tips, Benefits, Maintenance, and More - Healthline - May 21st, 2022
- Robert Downey Jr. Is Investing In Vegan Meat to Help Save the Planet - The Beet - May 21st, 2022
- Annual "Shred Cancer" Event to Raise $50000 for Cancer Prevention Research - PR Web - May 21st, 2022
- 'I Could Have Full-Blown Cancer Right Now,' Says Former Health Educator to the Black Community, Urging Them to Get Screened for Colon Cancer -... - May 21st, 2022
- Anxious Reddit parents wonder just how bad the baby formula shortage is and when it will end - Yahoo Entertainment - May 21st, 2022
- Plant-based diet for beginners: transition away from animal products - Livescience.com - April 12th, 2022
- The Maximum Amount Of Tuna You Can Safely Eat Each Week - Tasting Table - April 12th, 2022
- What is Unpasteurized Juice and Is It Safe? - Healthline - April 12th, 2022
- GoI needs to constitute the FOPL regulations as per WHO standards - National Herald - April 12th, 2022
- What to know about THCV or 'diet weed,' claims it can suppress appetite - Insider - March 31st, 2022
- Zocdoc Announces, "A Year in Nutrition" - PR Newswire - March 31st, 2022
- Paleo vs Whole30: Differences and Similarities Explained - Greatist - March 31st, 2022
- Dairy Foods & Cheese That Pregnant Women Should Avoid - Food Poisoning News - March 31st, 2022
- Ramadan 2022: How to fast during Ramadan when you have diabetes - Hindustan Times - March 31st, 2022
- Oyster Nutrition and Health Benefits - Greatist - March 31st, 2022
- Proven Ways to Stop Inflammation Anywhere on Your Body Eat This Not That - Eat This, Not That - March 31st, 2022
- Girls Gone Strong | Women-Specific Health and Fitness Coaching - runnersworld.com - March 31st, 2022
- Best Probiotics of 2022 - The Most Effective Probiotic Supplements - Islands' Sounder - March 31st, 2022
- Dr. Bernstein Diet - Weight Loss for Women, Weight Loss ... - January 28th, 2022
- Feeding your rabbits - PDSA - January 28th, 2022
- 1,200 Calorie Diet Menu - 7 Day Weight Loss Meal Plan - January 28th, 2022
- Intermittent Fasting and Rheumatoid Arthritis: What to Know - Everyday Health - January 28th, 2022
- A Wealth of Health | Safely managing weight loss and gain - The Breeze - January 28th, 2022
- hammerhead shark | Diet, Size, & Facts | Britannica - January 28th, 2022
- Is Cardio Really Necessary for Fat Loss? Here's the Deal - Greatist - January 28th, 2022
- The Rock does fasted cardio, then eats 6 to 7 meals per day - Insider - January 28th, 2022
- Crohn's Cooking and Me: Air-fryer honey garlic chicken to kick off a year of healthier eating - The Courier - January 28th, 2022
- How to live longer: The spice which may ward off cancer and heart disease - study findings - Daily Express - January 28th, 2022
- Intermittent Fasting: How to Break Your Fast Diet Doctor - December 30th, 2021
- 55 Things You Can Safely Feed to Ducks - Fruits, Veggies ... - December 30th, 2021
- Squirrels: Diet, Habits & Other Facts | Live Science - December 30th, 2021
- Intermittent fasting: Can it treat or reverse diabetes? - Medical News Today - December 30th, 2021
- The 21 Best Weight Loss Tips of 2021, According to Experts Eat This Not That - Eat This, Not That - December 30th, 2021
- Everything You Need To Know About Antioxidants - Health Essentials from Cleveland Clinic - December 30th, 2021
- Green soup recipe for weight loss with lots of nutrition and health - Central Valley Business Journal - December 30th, 2021
- New year's resolutions for pets (and their people) - The County - December 30th, 2021
- 122 Organizations Transforming Food Systems in 2022 Food Tank - Food Tank - December 30th, 2021
- Is It Illegal Driving With Your Interior Light On In Tx and AR? - kkyr.com - December 30th, 2021
- Election year puts Kishida's ability to deliver in focus - Japan Today - December 30th, 2021
- How long does cooked chicken last in the fridge? How to tell whether to bin it or binge - woman&home - December 30th, 2021
- Can you safely enjoy the holidays? Experts offer COVID tips - newsconcerns - December 20th, 2021
- The Weight Loss Coffee Thats Breaking The Internet - Inter Press Service - December 20th, 2021
- Simple mathematics of weight loss: How many calories to eat, how many to burn - Times Now - October 25th, 2021
- Egg nutrition: 9 health benefits of eggs - Insider - October 25th, 2021
- Celiac disease: Symptoms, diagnosis and how to get over this food allergy - Economic Times - October 25th, 2021
- Sunday Bulletin Board: Did she really say shed bought some Yogi Bear stamps? Or was that her husbands Boo Boo? - TwinCities.com-Pioneer Press - October 25th, 2021
- This might just look like grass, but it has the power to absorb a load of our carbon emissions - CNN - October 25th, 2021
- Mechanical Soft Diet: Pros, Cons, and Who It's For - Healthline - October 14th, 2021
- Archaeologists uncover massive, 1,500-year-old winemaking complex in Israel - Yahoo News - October 14th, 2021
- Resistant starch: Definition, health benefits, and foods - Insider - October 14th, 2021
- Ankylosing spondylitis physical therapy: Can it help? - Medical News Today - October 14th, 2021
- The Ultimate Guide to Nutrition and Fitness During Menopause - Healthline - October 5th, 2021
- Health tips from Dr. Oz and Dr. Roizen for 10-5-21 - The Commercial Dispatch - October 5th, 2021
- Eating on Campus: What to Grab and Where to Go - DrexelNow - Drexel Now - October 5th, 2021
- Can diabetes be controlled through diet? Read this new study - Economic Times - October 5th, 2021
- The CDC Just Released Guidance For Another Pandemic Holiday Season - Scary Mommy - October 5th, 2021
- Trumbull County Combined Health District warn residents to stay safe during Halloween, trick-or-treating - WKBN.com - October 5th, 2021
- Keeping Kids Safe: Local expert warns of signs that social media is affecting kids mental health - WYTV - October 5th, 2021