Global Blood Therapeutics is a clinical-stage biopharmaceutical company dedicated to discovering, developing and commercializing novel therapeutics to treat grievous blood-based disorders with significant unmet need. The Company’s lead product candidate, GBT440, is being developed as an oral anti-polymerization therapy for sickle cell disease (SCD). GBT440 holds significant potential for SCD patients since it is being investigated as a mechanism-based and potentially disease-modifying therapeutic. In ongoing clinical development, the Company is evaluating the impact of GBT440 on abnormal polymerization of deoxy-hemoglobin, the underlying mechanism of red blood cell sickling. The ability to address SCD at a fundamental level of pathology provides the potential to directly treat the disease, rather than its symptoms.
GBT has a pipeline of research programs addressing additional important blood disorders. The Company is conducting preclinical research to identify and develop an oral therapy for hereditary angioedema (HAE), a debilitating disease with limited current treatment options. Additionally, Global Blood is using GBT440’s ability to increase the oxygen affinity of hemoglobin to explore therapeutic potential beyond SCD. The Company plans to develop GBT440 as a potential treatment for acute and chronic hypoxemic pulmonary disorders. Initially, Global Blood will focus on areas of significant unmet, including idiopathic pulmonary fibrosis (IPF) and acute respiratory distress syndrome (ARDS).