Global Blood Therapeutics (GBT) is a clinical stage biopharmaceutical company developing novel small molecule therapeutics to treat grievous, non-malignant blood-based conditions for which there are currently only limited therapy. 

GBT440, the company’s lead drug candidate, is a potential once daily oral prophylactic therapy for patients with sickle cell disease (SCD).  By disrupting the fundamental pathophysiology of SCD, GBT440 has the potential to halt red blood cell sickling and disease progression, which could fundamentally transform the lives of SCD patients.  A Phase I/II study is ongoing, with early proof of concept expected in 2H 2015.

GBT has a pipeline of research programs addressing additional important blood disorders.  The company is currently identifying small molecule leads for an orally administered prophylactic therapy for HAE.  Additionally, GBT scientists are developing novel hemoglobin modifiers for pulmonary disorders in which increased oxygen delivery is needed (e.g. acute respiratory distress syndrome).

GBT’s R&D programs are discovered internally based on combining our extensive expertise and capabilities in blood biology, medicinal chemistry and structural biology.

GBT was launched in 2012 with funding from leading healthcare investor Third Rock Ventures. In December 2014, the company completed a $48 million Series B financing including investments from Wellington Management Company LLP, RA Capital, Deerfield Management, Sabby Capital, Perceptive Life Sciences, an affiliate of Cowen Group and one undisclosed blue chip public investment fund.