valoctocogene roxaparvovec

30th Jul 2025

Plain language summary: 4-year outcomes from testing valoctocogene roxaparvovec gene therapy in people with hemophilia A

In healthy people, a protein called factor VIII (FVIII) helps blood to clot and prevents excessive bleeding. People with hemophilia A lack FVIII because a faulty F8 gene is giving the wrong instructions to the liver cells that make it. Valoctocogene roxaparvovec (ROCTAVIAN™) is a gene therapy designed to transfer working copies of the F8 gene into liver cells. This summary describes the GENEr8-1 study, which looked at how well valoctocogene roxaparvovec works for treating people with severe hemophilia A compared with their usual FVIII replacement therapy, and its safety. 134 men received valoctocogene roxaparvovec; results from the first 2 years are reported.

14th Nov 2023

Plain Language Summary of Publication article: the GENEr8-1 study of valoctocogene roxaparvovec gene therapy for hemophilia A