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Hemoglobinopathies - Devastating Blood Diseases
The Hallmark of SCD: Pain
Allogeneic SCT is the only Curative Therapy Currently available
Symptoms Arise as Hemoglobin Switches from Fetal to Adult
Our Approach Disrupts the BCL11A Erythroid Enhancer
GLP/Tox Studies Support CT2001 Safety
Outline of Clinical Study