unoparvovec

Unoparvovec (also known as alphaglucuronosyltransferasegene unoparvovec or GT-UGT1A1-AAV8-02) is an investigational adeno-associated virus (AAV) serotype 8-based gene therapy developed for the treatment of **Crigler-Najjar syndrome type I (CN-I)**. CN-I is a life-threatening genetic disorder caused by mutations in the *UGT1A1* gene, which results in a deficiency or complete absence of the enzyme uridine diphosphate glucuronosyltransferase 1A1. This deficiency prevents the conjugation of bilirubin, leading to toxic accumulation of unconjugated bilirubin in the blood and a high risk of neurological damage or death. Unoparvovec utilizes a recombinant AAV2/8 vector to deliver a functional human *UGT1A1* cDNA cassette specifically to hepatocytes, with expression driven by the liver-specific thyroxine-binding globulin (TBG) promoter. By restoring hepatic UGT1A1 activity, the therapy facilitates the enzymatic conversion of unconjugated bilirubin into its water-soluble conjugated form for excretion. This approach aims to achieve a sustained reduction in serum bilirubin levels, potentially eliminating the need for daily phototherapy or liver transplantation in pediatric patients.

Brand names

Alphacrigen

Other names

alphaglucuronosyltransferasegene unoparvovec