NVC-001

NVC-001 is a first-in-class, adeno-associated virus serotype 9 (AAV9)-based gene therapy developed by Nuevocor for the treatment of LMNA-related dilated cardiomyopathy (LMNA DCM), a rare and aggressive genetic heart disease caused by mutations in the LMNA gene. The condition leads to rapid progression toward end-stage heart failure and life-threatening arrhythmias. NVC-001 is designed using proprietary mechanobiology platforms (PrOSIA and CARRE) to selectively silence mutant LMNA expression in cardiac muscle cells, thereby reducing mechanical stress on the nucleus, restoring nuclear envelope integrity, and addressing the root mechanobiological cause of disease. Preclinical studies have shown that NVC-001 can significantly extend survival and preserve cardiac function in animal models. The drug is administered as a single intravenous infusion[1][2][3][5][6].

Other names

LMNA DCM gene therapy NuevocorLamin A/C dilated cardiomyopathy gene therapy Nuevocor