HDAd-EPOR base editor

HDAd-EPOR base editor is an experimental in vivo gene therapy candidate designed for the treatment of various forms of anemia, including anemia of chronic disease and renal anemia. Developed by researchers at the University of Washington, the therapy employs a helper-dependent adenoviral (HDAd) vector to deliver a base editor—typically an adenine base editor (ABE)—directly to hematopoietic stem cells (HSCs). The base editor is engineered to introduce a precise gain-of-function mutation into the erythropoietin receptor (EPOR) gene, often mimicking the truncating mutations observed in primary familial and congenital polycythemia (PFCP). This modification renders the EPOR hypersensitive to erythropoietin, thereby stimulating the production of red blood cells even under conditions of low EPO availability or inflammatory suppression. This approach aims to provide a durable, one-time treatment that bypasses the need for exogenous erythropoiesis-stimulating agents (ESAs).

Other names

HDAd-EPOR-BEHDAd-EPOR base editing vector