AAV1-Δ27-264CFTR

AAV1-Δ27-264CFTR is an experimental, unapproved recombinant adeno-associated virus (AAV) gene therapy designed to deliver a truncated version of the cystic fibrosis transmembrane conductance regulator (CFTR) gene lacking amino acids 27-264 (Δ27-264-CFTR). Developed primarily by academic researchers at Johns Hopkins University, this gene therapy utilizes an AAV1 capsid (sometimes packaged with AAV2 inverted terminal repeats, referred to as AAV2/1) to target the CFTR chloride channel pathway. The truncated CFTR protein acts via transcomplementation to interact with and increase the expression of endogenous CFTR (both wild-type and ΔF508), restoring chloride channel currents. It is being investigated primarily for the treatment of cystic fibrosis, with exploratory preclinical studies also demonstrating efficacy in reducing cyst size and number in models of autosomal dominant polycystic kidney disease (ADPKD) and autosomal recessive polycystic kidney disease (ARPKD).

Other names

AAV1-CFTRAAV-1-CFTRAAV 1-CFTRAAV1-del27-264CFTRAAV-1-del27-264CFTRAAV 1-del27-264CFTRAAV1-Δ27-264 CFTRAAV1-Δ27-264-CFTRAAV2/1-Δ27-264CFTR