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A recent study published in Nature Medicine has reported the first-year results from a Phase 1 clinical trial investigating gene therapy for congestive heart failure (CHF). The Christ Hospital was the leading site for participant enrollment in this trial, which was sponsored by AskBio Inc., a subsidiary of Bayer AG.
The trial, identified as NCT04179643, focused on evaluating the safety and preliminary effectiveness of the investigational gene therapy AB-1002. It included participants with New York Heart Association (NYHA) Class III non-ischemic heart failure with reduced ejection fraction (HFrEF). CHF affects an estimated 64 million people globally, and current treatments often fail to prevent high rates of mortality and morbidity.
“These unique initial studies, show promising results for patients with congestive heart failure that have limited options for treatment,” 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. “Heart Failure investigators around the world have been trying to understand what exactly goes wrong in the heart and weakens its pumping activity until it finally fails.”
According to the publication, no adverse events were linked to the gene therapy. Participants showed clinically meaningful improvements across several efficacy measures. The data also indicated that the novel vector used in AB-1002 was highly targeted to cardiac tissue when given as a single intracoronary injection.
AskBio expressed gratitude toward those who participated in or supported this research effort: "AskBio thanks the participants who volunteered for this important clinical trial, the sites that made this effort possible, and the skilled investigators who conducted this invaluable research and contributed to the scientific body of knowledge related to AB-1002."
"We believe there is a critical need to progress innovative therapies that target the root causes of congestive heart failure, so we’re pleased to see these data for AB-1002 published and shared with the scientific community via Nature Medicine, a high-impact peer-reviewed journal," said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. "We’re eager to further assess the safety and efficacy of AB-1002 in our ongoing Phase 2 trial, GenePHIT, which is currently enrolling in Canada, Europe, the United Kingdom, and the United States, and look forward to sharing those results once available."
The ongoing GenePHIT Phase 2 trial is designed as an adaptive randomized double-blind placebo-controlled study examining both safety and efficacy outcomes for AB-1002 among patients with non-ischemic heart failure.
The Christ Hospital Health Network operates an acute care hospital in Mt. Auburn along with other facilities throughout its region. It employs more than 1,300 physicians and over 7,200 staff members. In 2025 it was recognized by U.S. News & World Report as Cincinnati’s top hospital; it also appeared on Newsweek’s World’s Best Hospitals list.
