tefidsogene civaparvovec

Tefidsogene civaparvovec (SB-913) is an *in vivo* genome editing therapy developed by **Sangamo Therapeutics** for the treatment of **Mucopolysaccharidosis II** (MPS II, also known as Hunter syndrome). The therapeutic utilizes a recombinant adeno-associated virus serotype 2/6 (rAAV2/6) vector system to deliver three components: two Zinc Finger Nucleases (ZFNs) and a donor DNA template encoding the human **iduronate 2-sulfatase (IDS)** enzyme. The ZFNs are engineered to target and create a double-strand break at a specific site within the **albumin (ALB)** gene locus in hepatocytes. Through homology-directed repair, the IDS transgene is integrated into this site, leveraging the highly active albumin promoter to drive the continuous production and secretion of the IDS enzyme into the systemic circulation. This approach is designed to provide a permanent, endogenous source of the enzyme, potentially eliminating the need for lifelong weekly enzyme replacement therapy (ERT) infusions.

Other names

tefidsogene civaparvovecSB-913SB913SB 913