First Patient Randomized in Phase II Gene Therapy Trial for Congestive Heart Failure


Phase II GenePHIT trial is evaluating the safety and efficacy of a single intracoronary infusion of AB-1002.

Image Credit: © photon_photo -

Image Credit: © photon_photo -

Bayer AG and Asklepios BioPharmaceutical (AskBio) announced that the first patient in the GenePHIT (Gene PHosphatase Inhibition Therapy) clinical trial has been randomized, according to a company press release.1

GenePHIT (NCT#05598333) is a Phase II trial of AB-1002 (also known as NAN-101), an investigational gene therapy, for the treatment of congestive heart failure (CHF). The trial is an adaptive, double-blind, placebo-controlled, randomized, multicenter study to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms who have been medically stable for at least four weeks.

According to the Heart Failure Society of America, current estimates show that nearly 6.5 million Americans over the age of 20 have heart failure. It directly accounts for about 8.5% of all heart disease deaths in the United States.2

“The randomization of the first patient as a part of the Phase II GenePHIT trial is an important moment for the heart failure community,” said Timothy D. Henry, MD, MSCAI, principal investigator and steering committee member, in the press release. “GenePHIT will evaluate the safety and efficacy of AB-1002 in the largest number of patients to date and improve our understanding of gene therapy overall for the treatment of congestive heart failure. The initiation of this trial brings us a step closer to potentially changing the course of this deadly and devastating disease.”

GenePHIT will include between 90 and 150 adults with left ventricular ejection fraction between 15% and 35%, who continue to suffer from heart failure symptoms despite guideline recommended therapy.

“Being able to announce this important GenePHIT trial update during Heart Failure Awareness Week adds special significance to this milestone,” said Roger J. Hajjar, MD, scientific chair CHF, AskBio, in the release. “The enrollment of this first patient in the Phase II trial represents the culmination of many years of dedicated research and development in all aspects of cardiac gene therapy for congestive heart failure. Although there is still much to learn about this early-stage investigational gene therapy, we hope today’s announcement, which highlights AskBio’s ability to advance AB-1002 gene therapy for the treatment of congestive heart failure, is encouraging news for everyone hoping to see new treatment options.”

“Heart failure is a devastating disease with increasing unmet medical need, especially in a progressively aging population,” said Christian Rommel, PhD, member of the executive committee of Bayer’s pharmaceuticals division and head of research and development in the release. “The potential impact of gene therapy to address this disease at its root cause is immense, and we are thrilled about this step in our path to deliver truly innovative treatment options for patients.”


  1. First Patient Randomized in AskBio Phase II Gene Therapy Trial for Congestive Heart Failure. News release. February 16, 2024. Accessed February 20, 2024.
  2. Heart Failure Facts & Information. Heart Failure Society of America. Accessed February 20, 2024.,is%20also%20a%20major%20killer.
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