volrubigene ralaparvovec

Volrubigene ralaparvovec is an investigational gene therapy that uses an adeno-associated virus serotype 8 (AAV8) vector to deliver a functional copy of the UGT1A1 gene. The therapy is designed for the treatment of Crigler-Najjar syndrome, a rare inherited disorder characterized by severe unconjugated hyperbilirubinemia due to mutations in the UGT1A1 gene. By introducing a working version of this gene into patients' liver cells, the therapy aims to restore normal bilirubin metabolism. Volrubigene ralaparvovec was initially developed by Audentes Therapeutics and later by Astellas Gene Therapies following acquisition[2][3]. Its development for Crigler-Najjar syndrome reached phase 1/2 clinical trials but was discontinued[3][4].

Other names

volrubigene ralaparvovec