CINCINNATI (November 14, 2023) – The Christ Hospital Heart & Vascular Institute has performed a first-in-human clinical trial using AB-1002 gene therapy for congestive heart failure patients.
AB-1002 is a one-time gene therapy administered to the heart to help promote increased production of a therapeutic protein inhibitor designed to block the action of protein phosphatase linked to congestive heart failure. The study sought to establish safety and preliminary efficacy of gene therapy AB-1002 (also known as NAN-101) in patients with advanced heart failure.
“Although current management has improved survival in heart failure patients, most therapies do not treat the underlying causes,” said Timothy D. Henry, MD, MSCAI, at The Christ Hospital Health Network, Principal Investigator and Steering Committee Member. “Consequently, the current standard of care does not reverse the trajectory of the disease to ultimate end-stage heart failure and death. These results show delivery of AB-1002 was well tolerated and resulted in positive efficacy outcomes in some patients with congestive heart failure.”
The initial results were encouraging. None of the patients in the trial showed adverse effects that were related to the study treatment. You can read more on the findings from the study here.
“These early results in patients living with advanced heart failure are encouraging as a possible treatment option for the future,” said Eugene Chung, MD, advanced heart specialist at The Christ Hospital Health Network. “Seeing the potential of gene therapy being explored in heart failure is an important step forward in one day potentially changing the direction of this devastating disease, which is a leading cause of morbidity and mortality in the United States.”
Heart failure happens when the heart is damaged and cannot pump enough blood and oxygen to support other organs in your body. Congestive heart failure results in the slowing of the blood flow out of the heart, which causes the blood returning to the heart through the veins to back up. This causes congestion in the body's tissues. Approximately 26 million people worldwide are living with congestive heart failure. This investigational gene therapy was made possible by Asklepios BioPharmaceutical, Inc. (AskBio).
About The Christ Hospital Health Network
The Christ Hospital Health Network consists of an acute care hospital located in Mt. Auburn, a remote hospital location in Liberty Township, five ambulatory outpatient centers and dozens of medical offices throughout the region. For more than 130 years, The Christ Hospital has provided compassionate care to those it serves. Made up for more than 1,300 physicians and more than 6,500 team members, our mission is to improve the health of our community by providing exceptional outcomes in an affordable way. The Network was recognized by U.S. News & World Report as the #1 hospital in the Cincinnati Region, named to Newsweek’s World’s Best Hospitals list in 2023. It is also a Press Ganey Guardian of Excellence Award recipient, which recognizes top-performing healthcare organizations that achieve the 95th percentile or above of performance in patient experience.
About AskBio
Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for congestive heart failure, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. An early innovator in the gene therapy field, with over 900 employees in five countries, the company holds more than 800 patents and patent applications in areas such as AAV production and chimeric capsids.