FBX-201

FBX-201 is an investigational adeno-associated virus (AAV) gene therapy program originally developed by Forge Biologics for a rare monogenic congenital disease, with limited public detail available on its specific target gene or indication. Reported as one of Forge’s initial in vivo AAV pipeline candidates, FBX-201 is described only at a high level as a gene therapy for a rare monogenic disease within the broader area of congenital disorders, without disclosed mechanism of action, target tissue, or clinical-stage data. As of the most recent public information, the program’s global development status is listed as pending, suggesting it remains in preclinical or early translational stages without an active registered clinical trial.