305,000. That's the number of births affected by Sickle cell disease (SCD) in 2010, making it the most common genetic hematological disorder. Millions of people are currently affected across the globe, the majority in low and middle income countries. The condition can cause acute pain, life-threatening infections, and other complications such as stroke or loss of vision. The disease can interfere with many aspects of the patient's life, including education, employment and psychosocial development resulting in significant socio-economic impact. Gene therapy as a potential cure for SCD has long been pursued, but so far with limited success. The Novartis Institutes for Biomedical Research (NIBR), the innovation engine of Novartis, with full funding support from the Bill and Melinda Gates
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