HCM

Disease Overview

Hypertrophic Cardiomyopathy (HCM) is a genetic disorder characterized by left ventricular hypertrophy and one of the most common forms of genetic cardiomyopathy. Mutations in over a dozen genes encoding the thick and thin filaments of the cardiac sarcomere have been linked to HCM. TNNI3, a critical component of the thin filament of the sarcomere, accounts for approximately 5% of all HCM cases.

Genetic mutations may affect any portion of the sarcomere, including thin and thick filaments, ultimately leading to cardiac muscle dysfunction. While thick filament mutations are the most common and best characterized, thin filament mutations remain clinically relevant. Some evidence suggests thin filament mutations are associated with more arrhythmias and earlier progression to advanced heart failure.

LX2022 Mechanism

LX2022 is a gene therapy candidate we are developing to deliver a functional TNNI3 gene to myocardial cells, to treat a distinct form of HCM due to mutations in the TNNI3 gene.