The Curative Track — Gene Therapy Centre
Breaking the cycle of lifelong blood transfusions by providing a one-time genetic cure—the global gold standard for Thalassemia treatment.
Vision
To break the cycle of lifelong transfusions by offering a one-time genetic cure. Gene therapy represents the gold standard for Thalassemia treatment and offers the opportunity to transform outcomes for thousands of affected children.
The Gene Therapy Centre of Excellence
We will build a state-of-the-art facility designed to meet international biosafety and clinical standards.
Planned infrastructure:
- Advanced Hematology Unit
- Pediatric Intensive Care Unit (PICU) for post-procedure monitoring
- Molecular & Genetic Laboratory for gene modification and processing
- Isolation-capable inpatient rooms
- International biosafety compliance
- Comprehensive digital registry to identify eligible candidates based on rigorous clinical criteria
Patient Journey
From registration to long-term cure:
- Registration & eligibility screening — Identification and assessment against clinical criteria.
- Genetic evaluation & counseling — Detailed genetic work-up and informed consent.
- Gene therapy administration — One-time treatment in a controlled setting.
- Post-procedure monitoring — PICU and specialist follow-up.
- Long-term cure & follow-up — Elimination of transfusion dependency and ongoing care.
Impact
- Elimination of transfusion dependency
- Prevention of iron overload, liver failure, and heart failure
- Restoration of childhood, education, and family stability
- Long-term economic relief for families