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12:00 AM
 | 
Feb 02, 2015
 |  BioCentury  |  Emerging Company Profile

Battling blood

How Global Blood's GBT440 could prevent RBC sickling in sickle cell disease

Global Blood Therapeutics Inc. is developing a small molecule that allosterically binds to hemoglobin and could be a disease-modifying treatment for sickle cell disease.

The product could provide an alternative to the only cure - bone marrow transplant - as well as to Phase III candidates that are seeking to reduce the duration or frequency of vaso-occlusive crisis. These acute pain crises are caused when sickled red blood cells block vessels, and usually require hospitalization and narcotics.

"We're not trying to treat crisis. We're trying to stop the disease," CEO Ted Love told BioCentury.

Sickle cell is caused by a point mutation in the beta globin gene that leads red blood cells to make abnormal hemoglobin, which polymerizes when deoxygenated, distorting the cells. Global Blood's GBT440 is an allosteric modifier that binds to the alpha chain of the abnormal hemoglobin to make it prefer the oxygenated state.

The company presented in vitro data at the American Society of Hematology meeting in December showing that GBT440 prevented RBC sickling in whole blood from...

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