AAV-TNNI3 gene therapy

AAV-TNNI3 gene therapy (LX2020) is an adeno-associated virus (AAV) vector-based gene therapy being developed by Lexeo Therapeutics for the treatment of genetic cardiomyopathies caused by mutations in the TNNI3 gene. The therapy utilizes an AAV9 capsid to deliver a functional human TNNI3 gene, which encodes cardiac troponin I, a critical component of the cardiac thin filament involved in muscle contraction. By restoring or supplementing the levels of functional troponin I in myocardial cells using a cardiac-specific promoter (such as cTnT), the therapy aims to address the underlying cause of hypertrophic cardiomyopathy (HCM), restrictive cardiomyopathy (RCM), and dilated cardiomyopathy (DCM) associated with TNNI3 mutations. Preclinical studies in porcine and murine models have demonstrated improved cardiac morphology, function, and survival, providing a translational foundation for clinical evaluation.

Other names

AAV-TNNI3AAV-TNNI-3AAV-TNNI 3TNNI3 gene therapyTNNI-3 gene therapyTNNI 3 gene therapy