Global Blood Therapeutics, Inc. is a clinical-stage biopharmaceutical company. The Company is engaged in discovering, developing and commercializing therapeutics to treat blood-based disorders. The Company is developing its initial product candidate, GBT440, as an oral, once-daily therapy for sickle cell disease (SCD). The Company is evaluating GBT440 in SCD in a Phase III clinical trial of GBT440 in adult and adolescent patients with SCD. GBT440 inhibits abnormal hemoglobin polymerization, the underlying mechanism of RBC sickling. The Company is also engaged in other research and development activities targeted towards hereditary angioedema (HAE). The Company’s GBT440 is an investigational drug that increases hemoglobin’s affinity for oxygen by binding to the alpha-chain of hemoglobin. GBT440 has been observed to keep a proportion of sickle hemoglobin (HbS), in its oxygenated state, where it cannot participate in polymerization.
廖博士：感觉这个药不错呀，不然FDA也不会这麽快提前通过了。First in class, 市场也大，美国就有十万病人。估计病人得终生服药，有利制药公司。最大的潜在威胁来自基因治疗药物的研发，不过，那应该在5年之后的事了
Q3 2019(9/30/19): Cash 603M. Loss 65M
EPS and Sales:
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Total institutions: 252，no change
Shares hold: 60322.5k shares. no change
shares% hold: 100%，no change
2019.11.30：Global Blood Therapeutics: 高确定性成长的罕见病公司
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2019.11.25: FDA Approves Oxbryta™ (Voxelotor), the First Medicine Specifically Targeting the Root Cause of Sickle Cell Disease
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