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RT-100 is a first-in-class, single-dose gene therapy candidate developed for the treatment of heart failure with reduced ejection fraction (HFrEF). The therapy consists of an inactivated adenovirus vector encoding human adenylyl cyclase type 6 (Ad5.hAC6), which is delivered directly into the coronary arteries during cardiac catheterization. Adenylyl cyclase type 6 (AC6) is a protein that regulates heart function and is often down-regulated in patients with heart failure. By increasing AC6 expression in cardiac tissue through gene transfer, RT-100 aims to improve cardiac contractility and overall heart function. Clinical studies have shown that a one-time administration of RT-100 can safely enhance heart performance beyond standard therapies and reduce hospitalization rates for heart failure. The drug has received Fast Track designation from the FDA due to its potential to address significant unmet needs in treating chronic heart failure[1][3][4][5][6].
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