We are developing adeno-associated viral (AAV) gene therapy-based treatments for genetically defined cardiomyopathies.
Dilated cardiomyopathy (DCM) is a serious unmet medical need, with a prevalence of approximately 1 in 250. It is one of the most common causes of heart failure, characterized by enlargement of the left ventricle, reducing the ability of the heart to pump blood.
The standard of care for DCM, involving the prescription of pharmacological inhibitors or implantation of devices, is unsatisfactory - serving mainly to delay disease progression. Currently, the only cure is a heart transplant.
While the cause for most DCMs is unknown, many are genetically-linked rare diseases, for which a gene therapy approach ought to be viable.
Nuevocor’s lead program is a recombinant adeno-associated virus (AAV)‑based gene therapy for patients suffering from DCM due to mutations in the Lamin A/C (LMNA) gene.
LMNA mutations are the #2 cause of familial DCM, affecting approximately 60,000 people in EU and USA. The mutation confers amongst the worst prognosis of all DCM, with increased risk of arrhythmogenic DCM and sudden cardiac death.
Since LMNA DCM is an autosomal dominant disease characterized by gain-of-function of the mutant Lmna protein, conventional gene replacement therapies would be ineffective. Nuevocor’s innovative approach to gene therapy circumvents this roadblock to treatment of LMNA DCM.
Using our novel target discovery platform, we are actively working on the treatment of additional genetically defined cardiomyopathies. We will bring to bear our deep expertise in cardiac genetics and physiology, mechanobiology, and viral vector technology to develop first-in-class gene therapies for the benefit of patients suffering from genetic heart disease.