A New Dawn for Hemophilia A? Lentiviral Gene Therapy Using Autologous Stem Cells

An innovative approach of delivering gene therapy via hematopoietic stem cells could revolutionize hemophilia A treatment by providing a one-time intervention with lasting effects.

The hemophilia treatment landscape is evolving with the approval of adeno-associated virus (AAV) vector-based gene therapies, but limitations underscore the need for better strategies. In a phase 1 trial conducted in India, investigators evaluated a novel gene therapy for severe hemophilia A in five participants aged 22 to 41 who received on-demand treatment and had an annualized bleeding rate (ABR) of at least 20 events. Investigators used autologous hematopoietic stem cells (HSCs) transduced with CD68-LV-ET3, a lentiviral vector containing a novel F8 transgene and a myeloid-directed CD68 promoter. Participants underwent myeloablative conditioning with treosulfan instead of busulfan. Therapy was administered with or without a transduction …

Reviewing Author

Anjali A. Sharathkumar, MBBS, MD, MS

Disclosures

Consultant/Advisory BoardGenentech

Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine

Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute

Anjali A. Sharathkumar, MBBS, MD, MS

Disclosures

Consultant/Advisory BoardGenentech

Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine

Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute

A New Dawn for Hemophilia A? Lentiviral Gene Therapy Using Autologous Stem Cells

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Reviewing Author