AskBio’s AAV Gene Therapy for Heart Failure Shows Promising Safety and Efficacy Data

RESEARCH TRIANGLE PARK, N.C. — October 21, 2025 — AskBio Inc., a gene therapy company and subsidiary of Bayer AG, announced the publication of 12-month data from its Phase 1 trial of the investigational gene therapy AB-1002 in patients with congestive heart failure (CHF) in the peer-reviewed journal Nature Medicine.

The data confirm that the first-in-human trial successfully met its safety objectives and demonstrated encouraging early efficacy signals in participants with New York Heart Association (NYHA) Class III non-ischemic heart failure with reduced ejection fraction (HFrEF).

Phase 1 Results Support Safety and Cardiotropism

The Phase 1, non-randomized, dose-escalation trial (NCT04179643) was designed to evaluate the safety and preliminary efficacy of AB-1002. Heart failure affects an estimated 64 million people globally, underscoring the critical need for new treatments.

Key findings from the published data include:

• Safety Confirmed: No adverse events were deemed related to AB-1002, supporting the therapy’s safety profile when administered as a single intracoronary injection.
• Efficacy Signals: Clinically meaningful improvements were recorded across several efficacy assessments in participants with non-ischemic CHF.
• Targeted Delivery: The data reinforce the finding that the AB-1002 capsid is highly cardiotrophic (heart-targeting), a crucial element for successful cardiac gene therapy.

“We believe there is a critical need to progress 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’re pleased to see these data for AB-1002 published and shared with the scientific community via Nature Medicine.”

About the Therapy and Next Steps

AB-1002 is a one-time gene therapy delivered directly to the heart. It is designed to promote the production of a modified therapeutic inhibitor (I-1c) protein. This protein works by blocking the action of protein phosphatase 1, an enzyme linked to the pathology of CHF.

The company is now focused on the next stage of development:

• Phase 2 Trial: The Phase 2 GenePHIT trial is currently enrolling participants in the United States, Canada, Europe, and the United Kingdom.
• Trial Design: GenePHIT is an adaptive, randomized, double-blind, placebo-controlled, multi-center trial that will further investigate the safety and efficacy of AB-1002 in patients with non-ischemic heart failure.