NEWS – Indian Scientists Develop Novel Gene Therapy for Haemophilia

HIGHLIGHTS

• Research Focus: Gene therapy for severe haemophilia A, a rare hereditary condition causing severe bleeding episodes +Does no use Adenovirus
• Research Success: Tested on five patients in Tamil Nadu; no bleeding episodes reported over 14 months.

Trial Details

• Lead Researcher: Alok Srivastava, Centre for Stem Cell Research (CSCR), Christian Medical College, Vellore.
• Funding: Union Department of Biotechnology.

CURRENT CHALLENGES IN HAEMOPHILIA TREATMENT

Conventional Treatments

• Frequent Injections: Requires repeated infusions of clotting factors.
• Severe Haemophilia A:
  • Characterized by less than 1% clotting factor.
  • Managed with:
    • Factor VIII replacement.
    • Monoclonal antibodies.
    • Substances mimicking Factor VIII.

Cost Implications

• High Costs: Estimated per-patient treatment cost in India over 10 years:
  • ₹2.54 crore (approx. $3,00,000).
• India’s Patient Pool:
  • Second-largest globally.
  • Estimated 40,000–1,00,000 patients.

GENE THERAPY

Benefits

• One-Time Treatment: Introduces a gene to produce clotting factors.
• Reduced Bleeding Incidents:
  • Example: Roctavian therapy reduced annual bleeding from 5.4 to 2.6 incidents.

Existing Gene Therapy

• Roctavian:
  • • FDA-approved in 2023.
    • Delivered using an adenovirus vector.
• Requires immune system suppression via corticosteroids.