4D-310 is an investigational genetic medicine candidate using the C102 vector that received Fast Track Designation from the FDA in Q3 2020 for the treatment of classic (severe) Fabry disease cardiomyopathy. 4D-310 is being evaluated in two Phase 1/2 INGLAXA clinical trials in adult patients with classic or late-onset Fabry disease. We believe this product is highly differentiated because it is designed for a single low dose intravenous delivery to, and transgene (GLA) expression within, the heart muscle cells (cardiomyocytes) in order to directly correct the leading cause of death in these patients (cardiomyopathy). Of note, cardiac (heart) benefit has not been definitively demonstrated following enzyme delivery in the blood through enzyme replacement therapy (ERT), PEGylated ERT, or gene therapy with enzyme expression from the liver; cardiac disease remains the leading cause of death in Fabry disease patients despite widespread use of ERT and related therapies.
