SAN DIEGO -- Results from a small single-center study showed that treatment with a gene therapy involving the use of lentiviral vector-transduced autologous hematopoietic stem cells (HSCs) increased factor VIII levels in patients with severe hemophilia A.

Current treatments require frequent Factor VIII replacement therapy, which faces challenges such as high costs, venous access in children, and low patient acceptance.

In the groundbreaking study the researchers observed an annualised zero bleeding rate in all the five subjects enrolled with the production of Factor VIII for a prolonged period of time thus obviating the need for repeated infusions.

Haemophilia is a severe bleeding disorder caused by the deficiency of clotting Factor VIII, leading to spontaneous internal and external bleeding episodes

India has successfully conducted its first in-human gene therapy for hemophilia A, with patients experiencing no bleeding episodes post-treatment.

Indian scientists successfully use gene therapy to treat severe haemophilia A, a rare hereditary condition with promising results.