Union Minister Dr. Jitendra Singh announced on Wednesday that researchers at the Christian Medical College (CMC) Vellore had completed the nation’s first human clinical trial of gene therapy for hemophilia A (FVIII deficiency). At the “National Science Day 2024” event held at Vigyan Bhawan here, he made this statement.

Factor VIII (FVIII), a clotting protein, can be absent or faulty in hemophilia A, a genetic disorder that results in prolonged bleeding in the patient.

In order to express an FVIII transgene in the patient’s own hematopoietic stem cell, which will subsequently express FVIII from particular differentiated blood cells, the clinical trials employed a novel technology involving the use of a lentiviral vector.

With an estimated 1,36,000 people suffering from hemophilia A, India is home to the second-largest population of patients with the disease.

Still, there are currently only about 21,000 registered.

In India, about 80% of cases of hemophilia remain undiagnosed due to a lack of screening tools for blood clotting in many hospitals and medical facilities, which hinders the diagnosis of new cases. “The manufacturing of this vector will commence soon in India and proceed with further clinical trials,” said the minister.

The Department of Biotechnology, the Centre for Stem Cell Research, an InStem Bengaluru unit, in association with Emory University, US at Christian Medical College, Vellore, provided support for the trial.

About 80% of haemophilia cases present with bleeding, usually affecting the big joints such as the knees, ankles, elbows, hips, and wrists, followed by deep bleeds that affect the large muscles of the body.

The most hazardous ones, however, are those that affect the neck, chest cavity, abdomen, pelvis, and head/brain.

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