Nuevocor has achieved a major milestone with the U.S. FDA’s clearance of its Investigational New Drug (IND) application for NVC-001, a first-in-class gene therapy targeting LMNA-related dilated cardiomyopathy (LMNA DCM)—a severe, life-threatening genetic condition. Developed using the company’s proprietary PrOSIA mechanobiology platform, NVC-001 is an AAV-based gene therapy uniquely designed to address the mechanical dysfunction at the root of the disease, specifically the loss of nuclear envelope integrity caused by LMNA gene mutations. This mechanobiological insight marks a significant shift in therapeutic strategy for cardiomyopathies, aiming to go beyond symptom management and modify the disease at a cellular level.

The upcoming Phase 1/2 first-in-human clinical trial will evaluate the safety, tolerability, and preliminary efficacy of NVC-001 in adults with LMNA DCM. Administered as a one-time intravenous infusion, the gene therapy is engineered to reduce pathological mechanical stress on cardiac cell nuclei and restore structural integrity. Preclinical studies have demonstrated promising results, including improved cardiac function and survival, supporting NVC-001’s potential as a disease-modifying treatment. For a condition with no approved targeted therapies and limited treatment options, this trial offers new hope for patients facing progressive heart failure.