ROCKVILLE, Md., Oct. 13, 2020 /PRNewswire/ --Foundation to Fight H-ABC, a non-profit organization dedicated to increasing awareness and driving development of a cure for the degenerative children's disease, H-ABC, today announced a sponsored research agreement with the University of Massachusetts Medical School and Yale University to advance a targeted gene therapy for H-ABC.
"We have high hopes to quickly prove efficacy with this approach to move research forward and find a permanent cure for this devastating disease," said Michele Sloan, Co-Founder, Foundation to Fight H-ABC.
H-ABC (hypomyelination with atrophy of the basal ganglia and cerebellum) belongs to a group of conditions called leukodystrophies, diseases that affect the white matter of the brain. These diseases disrupt the growth or maintenance of the myelin sheath, a protective layer that insulates nerve cells and allows for the transmission of messages between cells.
Caused by a mutation in the TUBB4A gene, H-ABC is a rare genetic disorder that affects certain parts of the brainspecifically the basal ganglia and the cerebellum, which control movement. H-ABC targets these important structures, reducing both their size and function. As a result, children who suffer from H-ABC often experience motor problems, cannot walk, talk, or sit on their own. Currently, there is no known cure for this disabling and life-threatening condition.
The teams of Dr. Guangping Gao (University of Massachusetts Medical School) and Dr. Karel Liem (Yale School of Medicine) will combine extensive expertise in the fields of Adeno-associated virus (AAV), a platform for gene delivery for the treatment of a variety of human diseases and H-ABC disease models, to develop AAV vectors to silence or outcompete the mutated TUBB4A gene.
"To date, AAV-based gene delivery system is the vector of choice for in vivo gene therapy of many currently untreatable rare diseases including H-ABC," said Guangping Gao, Ph.D. "We are very excited for starting close collaborations with Dr. Liem's team at Yale and the Foundation to Fight H-ABC to develop potential gene therapeutics for this devastating disease."
"With the support from the Foundation to Fight H-ABC, we are excited to build upon our mechanistic studies of the disease and to collaborate with Dr. Gao of the University of Massachusetts to develop and test AAV approaches to H-ABC," saidKarel F Liem Jr., M.D., Ph.D.
For more information, please visit https://www.h-abc.org/donate.
CONTACT: Sawyer Lipari, [emailprotected]
SOURCE Foundation to Fight H-ABC
See more here:
- After reeling back a gene therapy from Sanofi, a North Carolina upstart bags enough money to do something about it - Endpoints News - December 17th, 2020
- Gene Therapy in One Eye Improves Vision in Both Eyes - The Scientist - December 17th, 2020
- After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic - Endpoints News - December 17th, 2020
- Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,... - December 17th, 2020
- Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change - GlobeNewswire - December 17th, 2020
- Passage Bio Invests In Gene Therapy Manufacturing R&D Site - Contract Pharma - December 17th, 2020
- Gene Therapy Market Worth USD 35.67 Billion at 33.6% CAGR; Rising Prevalence of Spinal Muscular Atrophy to Augment Growth: Fortune Business Insights -... - December 17th, 2020
- Though Promising, Gene Therapies Face Durability And Reimbursement Headwinds - Forbes - December 17th, 2020
- What is gene therapy? - The Star Online - December 17th, 2020
- They thought their gene therapy failed. Instead, it spawned a medical mystery - Endpoints News - December 17th, 2020
- Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire - December 17th, 2020
- Gene Therapy Injection in One Eye Improves Vision in Both - Technology Networks - December 17th, 2020
- Locanabio Raises $100 Million to Advance RNA-Targeted Gene Therapies - BioSpace - December 17th, 2020
- Worldwide Gene Therapy Industry to 2025 - Cancer is Expected to Hold Significant Market Share in the Indication Segment - ResearchAndMarkets.com -... - December 17th, 2020
- Taysha Gene Therapies Set to Join Russell 2000 Index on December 21, 2020 - Business Wire - December 17th, 2020
- Rare Disease Gene Therapy Market: Increasing cases of genetic diseases to drive the market - BioSpace - December 17th, 2020
- Single gene therapy injection surprisingly boosts vision in both eyes - New Atlas - December 17th, 2020
- Freeline takes the haemophilia B gene therapy fight to Uniqure - Vantage - December 17th, 2020
- Gene Therapy for Hemophilia B Found Safe and Effective in First Phase III Trial - PRNewswire - December 14th, 2020
- ASH: UniQure/CSL hem B gene therapy curbs bleeding in phase 3even in patients with anti-AAV antibodies - FierceBiotech - December 14th, 2020
- Gene Therapy Market Analysis by Vector Type, Application, Region - Global Market Insights, Covid-19 Impact, Competition and Forecast to 2025 -... - December 14th, 2020
- Retinal Gene Therapy Market to Witness Sales Slump in Near Term Due to COVID-19; Long-term Outlook R - PharmiWeb.com - December 14th, 2020
- Gene Therapy, Absolutely and For Real | In the Pipeline - Science Magazine - December 14th, 2020
- Treatment with Investigational LentiGlobin Gene Therapy for Sickle Cell Disease (bb1111) Results in Complete Elimination of SCD-Related Severe... - December 14th, 2020
- Rocket Pharmaceuticals in orbit after gene therapy read-out - - pharmaphorum - December 14th, 2020
- Bluebird trumpets long-term data from beta-thalassaemia gene therapy - - pharmaphorum - December 14th, 2020
- ASH 2020: Novel Gene Therapy Found to be Safe and Effective in Treatment of Hemophilia B - OncoZine - December 14th, 2020
- Gene Therapy Unexpectedly Improves Vision In Both Eyes Of Patients Suffering A Form Of Blindness - IFLScience - December 14th, 2020
- Innovative payment models to support cell and gene therapies on the rise - MedCity News - December 14th, 2020
- Better education needed to give patients improved understanding of gene therapies, new review highlights - University of Birmingham - December 14th, 2020
- Global Gene Therapy Market Report 2020: Market is Expected to Recover and Reach $6.84 Billion in 2023 - Forecast to 2030 - GlobeNewswire - December 14th, 2020
- Navigating the Complexities of AAV Scale-Up and Manufacturing - Genetic Engineering & Biotechnology News - December 14th, 2020
- EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan - Business Wire - December 14th, 2020
- New CSL gene therapy could trigger a stock rerating - The Australian Financial Review - October 19th, 2020
- Cancer Gene Therapy Market Size & Share Insights on Growing Applications by 2026 - re:Jerusalem - October 19th, 2020
- Gene Therapy: Healing remedy or harmful hoax? The Knight News - The Knight News - October 19th, 2020
- Polyplus-transfection presents latest solution portfolio for gene therapy market to Alliance for Regenerative Medicine's Meeting on the Mesa -... - October 19th, 2020
- Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome - BioSpace - October 19th, 2020
- Merck's New VirusExpress Platform Speeds Development of Cell and Gene Therapies - PharmiWeb.com - October 19th, 2020
- Axovant Gene Therapies Receives Rare Pediatric Disease Designation for AXO-AAV-GM2 for Tay-Sachs and Sandhoff Disease - GlobeNewswire - October 19th, 2020
- Ori Biotech's new cell and gene therapy platform raises 23m - BusinessCloud - October 19th, 2020
- 5-Year-Old Girl, Being Treated In French Gene Therapy Trial, Dies In US - NDTV - October 19th, 2020
- Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire - October 19th, 2020
- Castle Creek Biosciences Announces First Patient Dosed in Phase 1/2 Clinical Trial of FCX-013 Gene Therapy for Treatment of Moderate to Severe... - October 19th, 2020
- Longview family rallies around 3-year-old with rare, fatal genetic condition - Longview News-Journal - October 19th, 2020
- Child dies in US during gene therapy trial - The News International - October 19th, 2020
- Tag: Gene Therapy - The Think Curiouser - October 19th, 2020
- Gene Therapy for Inherited Genetic Disorders Market Robust pace of Industry during 2018-2028 - The Think Curiouser - October 19th, 2020
- Disruptive Technologies and Mature Regulatory Environment Vital for Cell Therapy Maturation - BioSpace - October 19th, 2020
- Ori looks to advance closed cell therapy tech - BioProcess Insider - October 19th, 2020
- Histogen Appoints Moya Daniels as Executive Vice President and Head of Regulatory, Quality and Clinical Operations - BioSpace - October 19th, 2020
- EMA starts rapid review of Bluebird's gene therapy for rare disease CALD - - pharmaphorum - October 10th, 2020
- American Gene Technologies to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire - October 10th, 2020
- Unchained Labs launches the ultimate gene therapy tool; tackle AAV titer, empty/full ratio and more with Stunner! - PRNewswire - October 10th, 2020
- Smartphone-connected pacemaker devices, experimental gene therapy among top 10 innovations at annual event - BioWorld Online - October 10th, 2020
- CellGenix Completes Facility Expansion to Stay Ahead of the Increasing Demand for GMP Raw Materials - b3c newswire - October 10th, 2020
- Cleveland Clinic Names Top 10 Medical Innovations; Sickle Cell Therapy Tops the List - HealthLeaders Media - October 10th, 2020
- Investigational Gene Therapy for Phenylketonuria Gets Fast Tracked - Monthly Prescribing Reference - October 10th, 2020
- Flexion Therapeutics to Present at the 2020 Cell & Gene Virtual Meeting on the Mesa - GlobeNewswire - October 10th, 2020
- Taysha Gene Therapies Partners with Invitae to Enable Rapid Access to Genetic Testing and Earlier Diagnosis of Patients with CNS Disease for Rare and... - October 10th, 2020
- Cancer Gene Therapy Market 2020 by industry trends, statistics, key companies growth and regional forecast - News by Decresearch - October 10th, 2020
- Bone Therapeutics, Link Health and Pregene to develop and commercialize the ALLOB allogeneic bone cell therapy platform in China and Southeast Asia -... - October 10th, 2020
- Axovant to Participate in Upcoming October Conferences and Provide AXO-Lenti-PD Program Update the Morning of October 6th - GlobeNewswire - October 10th, 2020
- Investigational Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy - Monthly Prescribing Reference - October 10th, 2020
- Genprex to Present at the Alliance for Regenerative Medicine's Virtual Cell and Gene Meeting on the Mesa - Business Wire - October 10th, 2020
- Generation Bio to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire - October 10th, 2020
- Gyrolab AAVX Titer Kit Introduced to Support Cell and Gene Therapy Market - Technology Networks - September 2nd, 2020
- Taysha Gene Therapies Builds Experienced Executive Leadership Team to Advance Pipeline of Gene Therapies for Monogenic CNS Disease in Both Rare and... - September 2nd, 2020
- First Gene Therapy Products Approved in Brazil - Lexology - September 2nd, 2020
- Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40% - The News Brok - September 2nd, 2020
- Prevail Therapeutics to Present at Upcoming Investor Conferences - Yahoo Finance - September 2nd, 2020
- Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I - BioSpace - September 2nd, 2020
- The Safety and Clinical Activity of Gene-engineered T-cell Therapy Targeting HPV-16 E7 in Epithelial Cancers - Pharmacy Times - September 2nd, 2020
- AGTC to Host Management Update on September 9 at 8:00 am ET to Discuss Planned Phase 2/3 XLRP Trial, Share Additional XLRP Data and Report Fourth... - September 2nd, 2020
- Qualitative Analysis and Competitive Industry, CCOVID-19 Scenario of Personalized Gene Therapy Treatment Market - The News Brok - September 2nd, 2020
- Sorrento Announces the Closing of Its Acquisition of SmartPharm to Build Next Generation G-MAB-Encoded Plasmid DNA For Cost-Efficient and In Vivo... - September 2nd, 2020
- In the face of COVID-19, cell and gene therapy space shows 'remarkable resilience:' report - FierceBiotech - August 10th, 2020
- Gene Therapy Promising in BCG-Unresponsive Bladder Cancer - Medscape - August 10th, 2020
- Evolution and Expansion of Therapies in the Global Cell and Gene Therapy Tools and Reagents Market 2020-2024 - PRNewswire - August 10th, 2020
- How Gene Therapy Helped Conner Run : Short Wave - NPR - August 10th, 2020