CINCINNATI (November 10, 2025) - Nature Medicine, recently published the first-year results from its Phase 1 Trial using an investigational gene therapy in participants with congestive heart failure (CHF). The Christ Hospital was the top enroller for this clinical trial from AskBio Inc, a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG.
This non-randomized, sequential dose-escalation trial (NCT04179643) was designed to evaluate the safety and preliminary efficacy of investigational gene therapy AB-1002 in participants with New York Heart Association (NYHA) Class III non-ischemic heart failure with reduced ejection fraction (HFrEF). It is estimated that 64 million people worldwide are living with heart failure, and despite standard of care, mortality and morbidity remain very high.
“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.”
The publication, which is available online, confirms that no adverse events were related to Gene Therapy and that clinically meaningful improvements were recorded across multiple efficacy assessments in participants with non-ischemic CHF. The data further support that the novel vector used was highly cardiotropic when administered as a single intracoronary injection.
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."
GenePHIT is an ongoing Phase 2, adaptive, randomized, double-blind, placebo-controlled trial investigating the safety and efficacy of AB-1002 in non-ischemic heart failure.
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 135 years, The Christ Hospital has provided compassionate care to those it serves. Made up of more than 1,300 physicians and more than 7,200 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 2025.