AskBio Announces First Patient Dosed in Phase 1 Trial Using AAV Gene Therapy for Congestive Heart Failure

Research Triangle Park, North Carolina, UNITED STATES

RESEARCH TRIANGLE PARK, N.C. , Feb. 04, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical (AskBio), a clinical-stage adeno-associated virus (AAV) gene therapy company, and its NanoCor Therapeutics subsidiary today announced that the first patient has been dosed in a Phase 1 clinical trial of NAN-101. NAN-101 is a gene therapy that aims to activate protein phosphatase inhibitor 1 (I-1c) to inhibit the activity of protein phosphatase 1 (PP1), a substance that plays an important role in the development of heart failure.

Congestive heart failure (CHF) is a condition in which the heart is unable to supply sufficient blood and oxygen to the body and can result from conditions that weaken the heart muscle, cause stiffening of the heart muscles, or increase oxygen demand by the body tissues beyond the heart’s capability.

"Dosing the first patient using gene therapy to target I-1c to improve heart function is a tremendous milestone not only for the AskBio and NanoCor teams but, more importantly, for patients whose quality of life is negatively affected by CHF,” said Jude Samulski, PhD, Chief Scientific Officer and co-founder of AskBio. “We initially developed this gene therapy as treatment for late-stage Duchenne muscular dystrophy patients who typically die from cardiomyopathy. Following preclinical studies, we observed that heart function improved, which led us to investigate treatment for all types of heart failure.”

“We’re excited to be involved in this novel approach for patients with Class III heart failure,” said Timothy Henry, MD, FACC, MSCAI, Lindner Family Distinguished Chair in Clinical Research and Medical Director of The Carl and Edyth Lindner Center for Research at The Christ Hospital in Cincinnati, Ohio, and principal investigator for the study. “These patients currently have no other options besides transplant and left ventricular assist devices (LVAD). Today, we started to explore the potential of gene therapy to change their outcomes.”

Heart disease is the leading cause of death worldwide, with CHF affecting an estimated 1% of the Western world, including over six million Americans. There is no cure, and medications and surgical treatments only seek to relieve symptoms and slow further damage.

“Research by many investigators around the world has been trying to understand what exactly goes wrong in the heart and weakens its pumping activity until it finally fails,” said Evangelia (Litsa) Kranias, PhD, FAHA, Hanna Professor, Distinguished University Research Professor and Director of Cardiovascular Biology at the University of Cincinnati College of Medicine. “The aim has been to identify potential therapeutic targets to restore function or prevent further deterioration of the failing heart. Along these lines, research on the role of I-1c started over two decades ago, and it moved from the lab bench to small and large animal models of heart failure. The therapeutic benefits at all levels were impressive. It is thrilling to see I-1c moving into clinical trials with the hope that it also improves heart function in patients with CHF.”  

About the NAN-101 Clinical Trial
NAN-CS101 is a Phase 1 open-label, dose-escalation trial of NAN-101 in subjects with NYHA Class III heart failure. NAN-101 is administered directly to the heart via an intracoronary infusion by cardiac catheterization in a process similar to coronary angioplasty, commonly used to deliver treatments such as stem cells to patients with heart disease. The primary objective of the study is to assess the safety of NAN-101 for the treatment of NYHA Class III heart failure, as well as assess the impact of this treatment on patient health as measured by changes in exercise capacity, heart function and other factors including quality of life.

AskBio is actively enrolling patients with NYHA Class III heart failure to assess three doses of NAN-101. Please refer to for additional clinical trial information.

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About The Christ Hospital Health Network
The Christ Hospital Health Network is an acute care hospital located in Mt. Auburn with six ambulatory centers and dozens of offices conveniently located throughout the region. More than 1,200 talented physicians and 6,100 dedicated employees support the Network. Its mission is to improve the health of the community and to create patient value by providing exceptional outcomes, the finest experiences, all in an affordable way. The Network has been recognized by Forbes Magazine as the 24th best large employer in the nation in the magazine’s “America’s 500 Best Large Employers” listing and by National Consumer Research as the region’s “Most Preferred Hospital” for more than 22 consecutive years. The Network is dedicated to transforming care by delivering integrated, personalized healthcare through its comprehensive, multi-specialty physician network. The Christ Hospital is among only eight percent of hospitals in the nation to be awarded “Magnet” recognition for nursing excellence and among the top five percent of hospitals in the country for patient satisfaction. For more than 125 years, The Christ Hospital has provided compassionate care to those it serves.

About AskBio
Founded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy company dedicated to improving the lives of children and adults with genetic disorders. AskBio’s gene therapy platform includes an industry-leading proprietary cell line manufacturing process called Pro10™ and an extensive AAV capsid and promoter library. Based in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. AskBio maintains a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications with a current clinical pipeline that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy type 2i/R9 and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). For more information, visit or follow us on LinkedIn.


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