India Pharma Outlook Team | Thursday, 30 October 2025
AskBio, a fully owned and independently operated subsidiary of Bayer AG, has shared positive 12-month data from its Phase 1 clinical trial of AB-1002, an experimental gene therapy for congestive heart failure (CHF).
The results published in Nature Medicine signify a major step in the progress of gene therapy as a viable treatment for chronic heart disease.
The first-in-human, dose-escalation study (NCT04179643) explored the safety and initial effectiveness of AB-1002 in patients with New York Heart Association (NYHA) Class III non-ischemic heart failure with reduced ejection fraction (HFrEF).
The data indicated that no adverse events related to the treatment were observed, and the patients showed clinically significant improvements in various efficacy parameters.
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Heart failure is the cause of more than 64 million people worldwide and, even with the existing treatment options, it is still the major cause of hospitalizations and deaths. AB-1002, which was given as a single intracoronary injection, exhibited a cardiotropic effect that opens the door to the possibility of the therapy actually addressing the root causes of heart failure and not just alleviating the symptoms.
“These results give us hope, and it is another confirmation when they see the light of day in Nature Medicine,” said Dr. Canwen Jiang, Chief Development Officer and Chief Medical Officer at AskBio. “These data are a great support for the further exploration of AB-1002 in the ongoing Phase 2 GenePHIT trial, which is currently open for enrollment in North America and Europe.”
The study emphasizes AskBio’s dedication to breakthrough gene therapy innovations for complicated cardiovascular diseases.
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