By Wayne Koberstein, Executive Editor, Life Science Leader magazine
Follow Me On Twitter @WayneKoberstein
Intertwining science, business, and social missions to make progress against sickle cell disease
Global Blood Therapeutics (GBT) is developing new therapeutics for blood disorders, chiefly sickle cell disease (SCD), while also rallying the SCD community to seek and receive treatment. In November 2019, with Oxbryta (voxelotor), the company won the first-ever FDA approval of a significant new drug for treating SCD. Oxbryta earned accelerated approval based on increased hemoglobin, which the disease severely depresses. Oxbryta also appears to reduce pain and lower the incidence of vaso-occlusive crises (VOCs). Another product, inclacumab, is in Phase 3 trials for preventing and reducing hospital admissions for VOCs. A third candidate, coded GBT021601, is in early clinical development to deter sickling and destruction of red blood cells (RCBs). Potential therapeutics are in discovery, offering new approaches to SCD. GBT is also exploring new treatments for other blood disorders, such as beta thalassemia.