New Delhi: Bringing new hope to approximately 1,36,000 patients in India affected by Hemophilia A, the Department of Biotechnology (DBT) and the Centre for Stem Cell Research (CSCR) at CMC Vellore have successfully conducted the country’s first human gene therapy using a lentiviral vector for severe Hemophilia A.
In a groundbreaking study, researchers reported a zero annualized bleeding rate among all five participants enrolled in the trial, with sustained production of Factor VIII over an extended period, eliminating the need for repeated infusions, according to a statement by the Department of Biotechnology (DBT).
The volunteers were monitored for six months following the gene therapy. Factor VIII activity was found to correlate with the vector copy number in their peripheral blood. Over a cumulative follow-up period of 81 months, all five participants maintained a zero annualized bleeding rate, the department noted.
DBT further explained that scientists from the Centre for Stem Cell Research (CSCR) developed an alternative method for restoring Factor VIII expression using a lentiviral vector instead of an adeno-associated viral (AAV) vector. Based on encouraging preclinical findings, a first-in-human clinical trial was conducted to assess the safety and feasibility of this gene therapy approach for severe Hemophilia A.
The trial involved five participants who were treated with autologous hematopoietic stem cells (HSCs) transduced with the lentiviral vector carrying the Factor VIII gene. This innovative approach demonstrated promising results, offering a potential long-term solution for patients with severe Hemophilia A.
What is Hemophilia A?
Hemophilia A is a genetic disorder characterized by a deficiency in Factor VIII, a protein essential for blood clotting. This condition leads to excessive bleeding, either spontaneously or after injuries, posing severe risks to patients. It predominantly affects males due to its X-linked inheritance pattern.
What is Gene Therapy?
Gene therapy involves modifying or replacing faulty genes within a patient’s cells to treat or prevent disease. For Hemophilia A, this approach focuses on introducing a functional version of the defective gene responsible for Factor VIII production.
