AAV-CFH

AAV-CFH is an investigational adeno-associated virus (AAV) gene therapy designed to deliver a complement factor H (CFH) transgene to ocular tissues in order to restore local regulation of the alternative complement pathway in the retina. CFH is a key inhibitor of alternative pathway complement activation, and loss or dysfunction of CFH contributes to complement overactivation and pathology in age-related macular degeneration and related conditions. Preclinical AAV-CFH-like constructs typically use truncated or otherwise size-optimized CFH transgenes packaged in AAV vectors and administered by subretinal or related intraocular routes to achieve sustained, local CFH expression and rescue of complement dysregulation in CFH-deficient models, supporting development for geographic atrophy and other dry AMD phenotypes.