ranuzifigene civaparvovec

Ranuzifigene civaparvovec is an investigational gene therapy that utilizes a recombinant, non-replicating adeno-associated virus (AAV) type 2/6 vector to deliver a therapeutic transgene. The vector expresses a cDNA targeting the albumin gene locus in hepatocytes, enabling targeted insertion and expression of the therapeutic gene. This approach is designed for in vivo genome editing or gene addition, with the goal of providing long-term correction of genetic disorders by enabling sustained production of the missing or deficient protein. Ranuzifigene civaparvovec belongs to a class of AAV-based gene therapies and shares its suffix ("civaparvovec") with other similar investigational agents. The primary developer associated with this drug is Sangamo Therapeutics. Based on its mechanism and class, it is likely being developed for rare genetic diseases requiring liver-directed gene addition.