Global Blood Therapeutics

Global Blood Therapeutics is focusing on changing the treatment paradigm for chronic, non-malignant blood diseases for which there are currently no effective cures and only extremely limited therapeutic options.

The current standard of care treats the symptoms during acute episodes; however, the underlying pathology of the disease causes progressive damage to the body. Global Blood Therapeutics is focused on the critical need for therapeutics that address the causes of blood diseases, not just the symptoms.

As one of the largest and most accessible organs, blood is a rich source of biologically and clinically validated targets. The Company is pursuing blood-based targets for which the biology is well understood but previously have been considered “undruggable”. Global Blood Therapeutics customizes its approach to successfully prosecute these targets based on the Company's unique insights or expertise.

Global Blood Therapeutics’ extensive drug discovery capabilities — combining world-class medicinal chemistry with deep expertise in blood biology — are driving its product pipeline of mechanism-based therapeutics. The Company brings together unique insights into protein structure and function, comprehensive biological assays and advanced drug discovery techniques. These foundational technologies are customized to specific disease targets and include:

Conformational modulation (changing the shape) of specific blood-based proteins
Allosteric activation and allosteric inhibition
Computational methods in protein structure, dynamics and ligand docking
Broad access to diverse libraries of compounds
Comprehensive biological assays ranging from purified protein to whole blood and genetically based animal models
The Company’s differentiated approach is enabling the development of novel, orally-available, potent compounds that ultimately should translate into high therapeutic efficacy and decreased toxicity. The Company's pipeline of preclinical programs is focused on several blood diseases, including sickle cell disease and hereditary angioedema.