Sickle cell disease poised for fresh infusion of hemoglobin-based endpoints
Global Blood's sickle cell candidate sets the stage for evaluating disease-modifying therapies using hemoglobin-based endpoints
As new therapies designed to cut off sickle cell disease pathology at the root advance through the clinic, the field is moving beyond endpoints centered on pain crises to readouts based on hemoglobin. A single, standardized hemoglobin endpoint seems unlikely, however, as different classes of disease-modifying candidates boost different versions of hemoglobin.
The first test will be Global Blood’s NDA for voxelotor, which hinges on boosting endogenous hemoglobin levels. But a negative result will have little read through to the next disease-modifying therapies in line, which rely on engineered or fetal forms of hemoglobin...
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