Nuevocor, a biotechnology company focused on genetically defined cardiac diseases, has announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for NVC-001, a novel gene therapy candidate for LMNA-related dilated cardiomyopathy (DCM).
LMNA-related DCM is a progressive and life-threatening condition caused by mutations in the LMNA gene, often leading to heart failure and life-threatening arrhythmias. Currently, there are no approved treatments that specifically target the underlying genetic cause.
NVC-001 is an AAV9-based gene therapy developed using Nuevocor’s proprietary CARRE (Cardiomyocyte-selective Allo-repressive RNA Expression) platform. The therapy aims to selectively silence the expression of the mutated LMNA gene in heart muscle cells, with the goal of halting disease progression and improving cardiac function.
In preclinical studies, NVC-001 demonstrated the ability to significantly extend survival and preserve heart function in mouse models of LMNA-related DCM. With the FDA’s clearance of the IND, Nuevocor plans to begin a Phase 1 clinical trial in adult patients later this year.
“This is a major milestone for our community,” said the team at LMNAcardiac.org. “We will be following the progress of this trial closely and sharing updates as they become available.”