Nuevocor Receives FDA IND Clearance for NVC-001, an AAV Gene Therapy for LMNA-Related Dilated Cardiomyopathy

SINGAPORE & PHILADELPHIA – June 10, 2025 – Nuevocor, a biotechnology company headquartered in Singapore and focused on developing cures for cardiomyopathies driven by aberrant mechanobiology, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for NVC-001. NVC-001 is an adeno-associated virus (AAV)-based gene therapy specifically designed to treat LMNA-related dilated cardiomyopathy (LMNA DCM). With this clearance, Nuevocor plans to initiate an open-label, ascending-dose Phase 1/2 clinical trial for NVC-001 in patients with LMNA DCM, marking a significant advancement in AAV gene therapy for cardiac conditions.

“This IND clearance marks a significant milestone in our mission to develop transformative therapies for patients with genetic cardiomyopathies by leveraging unique insights from our proprietary PrOSIA mechanobiology platform,” said Dr. Yann Chong Tan, co-founder and CEO of Nuevocor. “NVC-001 is the first disease-modifying therapy for LMNA DCM designed to address the underlying mechanobiological root cause of disease through its AAV delivery system. We look forward to initiating our clinical trial to bring this potentially life-changing therapy to patients.”

LMNA DCM is a serious genetic heart condition caused by mutations in the LMNA gene. This gene encodes lamin A/C, a vital protein for maintaining nuclear envelope integrity and regulating gene expression within cardiac cells. Mutations in LMNA lead to a weakening of the heart muscle, arrhythmias, and progression to end-stage heart failure. It is estimated to affect approximately 100,000 patients across the U.S. and Europe, representing a significant unmet medical need due to the lack of disease-modifying treatments.

NVC-001, an AAV gene therapy, is engineered to address the functional mechanical root cause of LMNA DCM by reducing aberrant mechanical stress on the nucleus, thereby restoring nuclear envelope integrity. Nuclear envelope disruption is a recognized hallmark of the disease. In preclinical studies, treatment with NVC-001 demonstrated significant benefits, including improvements in survival and cardiac function.

The planned Phase 1/2 clinical trial will be a first-in-human, 52-week, open-label, multicenter, ascending-dose study. Its primary objective is to evaluate the safety, tolerability, and preliminary efficacy of NVC-001 in adult subjects with LMNA-DCM. NVC-001 will be administered as a one-time intravenous infusion to patients in ascending-dose cohorts. This trial represents a crucial step toward bringing a potentially transformative AAV therapy to patients suffering from this debilitating genetic heart condition.