India’s First in human Gene Therapy for Hemophilia A

India’s first in Human Gene Therapy with lentiviral vector for Severe Hemophilia A in a single center study resulted in an annualised zero bleeding rate in all the 5 subjects enrolled with the production of Factor VIII for a prolonged period of time thus obviating the need for repeated infusions.

• Breakthrough:
  • Achieved zero bleeding events (annualized) in all five subjects after treatment.
  • Factor VIII — the deficient clotting protein in Hemophilia A — was produced for a prolonged period, eliminating the need for repeated infusions.
• How It Was Done:
  • Used lentiviral vector-based gene therapy.
  • Conducted as a single-center study by the Centre for Stem Cell Research (CSCR) at CMC Vellore, under the BRIC-inStem initiative.
  • Supported by India’s Department of Biotechnology.

About Hemophilia A:

• A serious bleeding disorder caused by a deficiency of clotting Factor VIII.
• Leads to spontaneous bleeding, joint damage, and reduced quality of life.
• Treatment Challenges:
  • Requires repeated, expensive infusions of Factor VIII.
  • Problems like high cost, difficult venous access (especially in kids), and low patient compliance.

India’s Hemophilia Burden:

• India has the world’s second largest number of Hemophilia cases:
  ➔ About 1,36,000 people living with the disorder.

(Source: PIB)