▎WuXiEdited by Kant Content Team

Recently, Bayer) Group and its wholly-owned independent operating subsidiary Asklepios BioPharmaceutical (AskBio) announced,The U.S. FDA has granted the AB-1002 project Fast Track designation.AB-1002 is an investigational one-time gene therapy administered to target the heart.Aimed at promoting the constitutively active form of protein inhibitor 1 (I-1c), blocking the action of protein phosphatase 1.Inhibiting the function of this protein associated with congestive heart failure (CHF) may have therapeutic effects.

When the heart cannot pump effectivelyHeart failure occurs when the blood meets the body's needs (including providing sufficient oxygen to the organs).Congestive heart failure can slow the flow of blood out of the heart, causing backflow when blood returns to the heart through the veins.This can lead to congestion in body tissues, with symptoms including swelling in the legs and ankles.Sometimes, fluid accumulates in the lungs and interferes with breathing.Approximately 26 million people worldwide suffer from congestive heart failure.

AB-1002 (also known as NAN-101) is an investigational gene therapy currently in patient recruitment for the Phase 2 clinical trial of GenePHIT (Gene Phosphatase Inhibition Therapy).GenePHIT is a Phase 2 adaptive, double-blind, placebo-controlled, randomized, multicenter trial designed to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in male and female patients aged 18 years and older with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms. Participants are randomized in a 1:1:1 ratio to receive low-dose, high-dose, or placebo treatment. The primary outcome measures include cardiovascular-related death, changes in NYHA class from baseline, left ventricular ejection fraction (LVEF), peak oxygen consumption (pVO2), and the 6-minute walk test (6MWT).

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AskBio, a wholly-owned and independently operated subsidiary of Bayer AG, is a company fully dedicated to gene therapy, committed to developing life-saving drugs and transforming patients' lives. The company has a range of clinical programs covering neuromuscular, central nervous system, cardiovascular, and metabolic disease indications, as well as a pipeline of products in clinical stages, including congestive heart failure, Huntington's disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson's disease, and Pompe disease.