Lentiviral vector (LV)–based gene therapy using CD34+ hematopoietic stem cells (HSCs) demonstrated stable factor VIII expression in severe hemophilia A patients, with higher vector copy numbers correlating to increased factor VIII activity.

Treatment assessment focused on safety endpoints including engraftment and regimen-related toxic effects, and efficacy endpoints including factor VIII activity and annualized bleeding rate. Participants demonstrated successful engraftment with median time to neutrophil engraftment of 11 days and platelet engraftment of 15 days. “Gene therapy for hemophilia A with the use of lentiviral vector-transduced autologous HSCs resulted in stable factor VIII expression, with factor VIII activity correlating to vector copy number in the peripheral blood,” wrote the authors of the study.

The study was supported by grants from the Department of Biotechnology, Ministry of Science and Technology, Government of India, and the National Institutes of Health. 

Source: MEDspace