4D-310

4D-310 is an investigational gene therapy developed for the treatment of Fabry disease, particularly Fabry disease cardiomyopathy. It uses a targeted and evolved adeno-associated virus (AAV) vector called C102 to deliver a functional copy of the GLA gene into patients' cells. This enables long-term production of alpha-galactosidase A (alpha-Gal A), the enzyme deficient in Fabry disease, thereby reducing harmful substrate accumulation such as globotriaosylceramide (Gb3). The therapy is designed for single intravenous administration and aims to achieve high local enzyme production in critical organs like the heart, blood vessels, and kidneys, as well as systemic cross-correction via sustained serum levels. Early clinical data show improvements in cardiac function with a generally favorable safety profile; however, some cases of atypical hemolytic uremic syndrome (aHUS) have been observed but resolved[1][2][5][6][8].