NAN-101 is an AAV gene therapy being developed to treat congestive heart failure. Credit: Arek Socha from Pixabay.

Asklepios BioPharmaceutical (AskBio) and its subsidiary NanoCor Therapeutics have dosed the first patient in a Phase I clinical trial of gene therapy candidate NAN-101 to treat congestive heart failure.

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Congestive heart failure is characterised by the inability of the heart to supply the necessary blood and oxygen to the body. The condition lacks cure, while drugs and surgical therapy are intended to alleviate symptoms and slow damage.

NAN-101 is an adeno-associated virus (AAV) gene therapy designed to stimulate protein phosphatase inhibitor 1 (I-1c) to block the action of protein phosphatase 1 (PP1), which is linked to heart failure.

The medication is directly given to the heart via an intracoronary infusion.

The NAN-CS101 open-label, dose-escalation, multi-centre trial will evaluate three doses of the gene therapy in approximately 12 patients with NYHA Class III heart failure.

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The primary objective of the study is safety, along with the effect of NAN-101 on patient health, which is tracked as changes in exercise capacity, heart function, and quality of life.

AskBio chief scientific officer and co-founder Jude Samulski said: “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.

“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.”

The Phase I trial is expected to be completed in July next year.

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