Updates
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Updates
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Monday, January 8, 2019
Crizanlizumab (SEG101) Receives FDA Breakthrough Therapy Designation
“Crizanlizumab is a monthly infusion under development to prevent pain crises (also called vaso-occlusive crises, or VOCs) in patients with sickle cell disease.”
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Monday, January 8, 2019
FDA Awarded Fast Track Status to CTX001 for Sickle Cell Disease
“CTX001 uses the CRISPR gene-editing technology to increase the production of fetal hemoglobin in patients’ red blood cells.” Sickle Cell Anemia News
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Monday, January 8, 2019
GBT Plans to Submit NDA after FDA Agrees with Accelerated Approval Pathway for Voxelotor
“Voxelotor is an oral, once-daily therapy for patients with SCD by increasing hemoglobin’s affinity for oxygen and blocks sickling of red blood cells.”
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