KHN921

KHN921 is a recombinant, replication-deficient adeno-associated virus serotype 9 (AAV9) gene therapy designed for the treatment of hypertrophic cardiomyopathy (HCM) associated with mutations in the MYBPC3 gene. Developed jointly by Chengdu Origen Biotechnology and Vanotech, the therapy utilizes an AAV9 vector to deliver a functional copy of the MYBPC3 gene directly to cardiomyocytes via a single intracoronary infusion. This approach aims to restore normal levels of cardiac myosin-binding protein C, addressing the underlying genetic cause of the disease. KHN921 has received FDA Investigational New Drug (IND) clearance to initiate a Phase I/II clinical trial in symptomatic adults with MYBPC3 mutation-associated HCM.