YAP101

YAP101 is a gene therapy designed for myocardial regeneration in patients with ischemic heart failure and reduced ejection fraction (HFrEF). It utilizes an adeno-associated virus serotype 9 (AAV9) vector equipped with a cardiomyocyte-specific promoter to deliver short hairpin RNAs (shRNAs) that target Salvador 1 (SAV1), a key regulator of the Hippo signaling pathway. By suppressing SAV1, YAP101 transiently inhibits the Hippo pathway, thereby promoting cardiomyocyte proliferation, reducing fibrosis, and aiming to restore heart function. This approach represents a novel regenerative strategy, directly targeting the molecular drivers of cardiac repair rather than simply treating symptoms. The most advanced clinical investigation is a Phase I single-center, open-label, dose-escalation trial in adults with ischemic HFrEF. Preclinical studies demonstrated improved ejection fraction and reduced scar size in a large animal model. The drug is delivered via a one-time transendocardial injection to the left ventricle and monitored for effect on heart function, biomarkers, exercise tolerance, and quality of life over 12 months[1][2][3][4][5].

Brand names

YAP101YAP-101YAP 101

Other names

AAV9-Sav-shRNAAAV-9-Sav-shRNAAAV 9-Sav-shRNA