Therapeutic Pipeline
Pathway-Specific
Genetic Medicines

NVC-001 is a first-in-class, AAV-based gene therapy candidate for the treatment of LMNA-related dilated cardiomyopathy (LMNA DCM), one of the most aggressive forms of  DCM with a rapid progression toward end-stage heart failure and malignant ventricular arrhythmias associated with increased risk of sudden cardiac death. LMNA DCM is estimated to affect more than 100,000 patients in the U.S. and Europe. NVC-001 is designed to reduce forces to the nucleus to restore nuclear envelope integrity, a hallmark of LMNA DCM, and treat disease. NVC-001 has demonstrated dramatic survival benefits in preclinical models, high transduction levels and a clean safety profile in both GLP toxicology studies and large animal models.