Oct. 21, 2025 – AskBio Inc., a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has announced the publication in peer-reviewed journal Nature Medicine of 12-month data from its Phase 1 trial of AB-1002 investigational gene therapy in participants with congestive heart failure (CHF).1
This non-randomized, sequential dose-escalation trial (NCT04179643) includes escalating dose cohorts 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).1 It is estimated that 64 million people worldwide are living with heart failure, and despite standard of care, mortality and morbidity remain very high.2,3
The publication, which is available online, confirms that no adverse events were deemed related to AB-1002 in this trial and that clinically meaningful improvements were recorded across several efficacy assessments in participants with non-ischemic CHF.1 The data further support that the AB-1002 capsid may be highly cardiotrophic 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 a Phase 2, adaptive, randomized, double-blind, placebo-controlled trial investigating the safety and efficacy of AB-1002 in non-ischemic heart failure.
AB-1002
AB-1002 is an investigational one-time gene therapy administered to the heart to promote production of a modified version of the therapeutic inhibitor 1 (I-1c) protein designed to block the action of protein phosphatase 1, which is linked to CHF.4,5 This investigational gene therapy has not been approved by any regulatory authority, and its efficacy and safety have not yet been established or fully evaluated.
References
- Hajjar, R. Cardiotropic AAV gene therapy for heart failure: a phase 1 trial. Nature Medicine. 2025 Oct 21; 10.1038/s41591-025-04011-z.
- Savarese G, et al. Global burden of heart failure: a comprehensive and updated review of epidemiology. Cardiovasc Res. 2023 Jan 18;118(17):3272-3287.
- Myhre P, et al. Digital tools in heart failure: addressing unmet needs. Lancet Digit Health. 2024 Oct;6(10):e755-e766.
- Henry T, et al. Preliminary safety and efficacy of a Phase 1 clinical gene therapy trial in patients with advanced heart failure using a rationally designed cardiotropic AAV vector targeting Protein Phosphatase Inhibitor-1. Presented at American Heart Association Scientific Sessions, November 2023.
- Nicolaou P & Kranias E. Role of PP1 in the regulation of Ca cycling in cardiac physiology and pathophysiology. Front Biosci (Landmark Ed). 2009 Jan 1;14(9):3571-85.
October 28, 2025 
