AAV8-cFGF23

AAV8-cFGF23 is a preclinical gene therapy construct designed for the treatment of X-linked hypophosphatemia (XLH). It consists of an adeno-associated virus serotype 8 (AAV8) vector engineered to deliver a transgene encoding the C-terminal fragment of fibroblast growth factor 23 (cFGF23) to liver hepatocytes, typically under the control of a liver-specific promoter such as human alpha-1-antitrypsin (hAAT). The expressed cFGF23 peptide acts as a competitive inhibitor of full-length, biologically active FGF23 by competing for binding to the FGF receptor/alpha-Klotho complex. By antagonizing excessive FGF23 activity, the therapy aims to restore phosphate homeostasis, normalize vitamin D metabolism, and correct skeletal mineralization defects. Research has also evaluated optimized versions, including those utilizing murine-specific sequences for preclinical modeling and Fc-fusion proteins to enhance stability and therapeutic potency.

Other names

AAV8-hAAT-cFGF23AAV-8-hAAT-cFGF23AAV 8-hAAT-cFGF23AAV8-cFGF23-FcAAV-8-cFGF23-FcAAV 8-cFGF23-Fc