AAV-hCFH

AAV-hCFH is a liver-directed gene therapy designed to treat Complement 3 glomerulopathy (C3G), a rare kidney disease caused by uncontrolled activation of the complement alternative pathway due to Factor H (CFH) mutations. This therapy utilizes an Adeno-Associated Virus (AAV) vector, specifically an AAV2 vector packaged into an AAV8 capsid, to deliver the human CFH gene under the control of a liver-specific promoter. The goal is to enable the liver to produce functional human CFH, thereby restoring complement regulation and mitigating kidney injury. Preclinical studies in a C3G mouse model demonstrated that AAV-hCFH successfully transduced the liver, increased serum CFH levels, reduced serum C3 levels, and improved kidney function, including reduced proteinuria and kidney injury.