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

In a significant medical feat, Indian scientists have developed the first-in-human gene therapy using lentiviral vectors for severe haemophilia A

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.

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