By: IPP Bureau
Last updated : October 29, 2025 11:47 am
Results demonstrate favorable safety profile and clinically meaningful improvements in patients with non-ischemic heart failure
AskBio, a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced the publication of 12-month data from its Phase 1 clinical trial of AB-1002, an investigational gene therapy for congestive heart failure (CHF), in the peer-reviewed journal Nature Medicine.
The first-in-human, non-randomized, sequential dose-escalation study (NCT04179643) was designed to evaluate the safety and preliminary efficacy of AB-1002 in participants with New York Heart Association (NYHA) Class III non-ischemic heart failure with reduced ejection fraction (HFrEF). The findings showed that no adverse events were attributed to AB-1002, and participants demonstrated clinically meaningful improvements across multiple efficacy assessments.
Heart failure affects an estimated 64 million people worldwide, and despite current standard-of-care treatments, it remains a leading cause of morbidity and mortality. The published data indicate that AB-1002, when administered as a single intracoronary injection, may exhibit strong cardiotropic properties and has the potential to address the underlying causes of heart failure.
“We believe there is an urgent need to advance innovative therapies that target the root causes of congestive heart failure,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. “We are encouraged by these Phase 1 results and pleased to see them published in Nature Medicine, a high-impact scientific journal. We look forward to further evaluating AB-1002 in our ongoing Phase 2 GenePHIT trial.”
The GenePHIT trial is a Phase 2, adaptive, randomized, double-blind, placebo-controlled study currently enrolling participants in Canada, Europe, the United Kingdom, and the United States. It aims to further investigate the safety and efficacy of AB-1002 in individuals with non-ischemic heart failure.
AskBio extends its gratitude to the trial participants, investigators, and clinical sites that made this research possible and contributed to advancing the understanding of gene therapy in heart failure.