The protein crystallography capabilities at the Advanced Light Source’s (ALS’s) Beamline 8.3.1 have been critical to Global Blood Therapeutics’ (GBT’s) ongoing effort to formulate a better treatment for sickle cell disease (SCD). The Bay Area biopharmaceutical company is developing a therapy that prevents the polymerization of hemoglobin, the key event in promoting the sickle shape and advancing the disease. Working closely with beamline scientist and Biosciences (MBIB) faculty James Holton, GBT obtained results from the ALS that enabled them to understand the molecular mechanism of their compound GBT440—which works by binding to hemoglobin and increasing the affinity for oxygen, thereby inhibiting polymerization—and gain insight into other potential molecular targets. Much of the research GBT conducts is confidential, but the company has already published two papers based on their work at the ALS. The compound GBT440 is currently in phase 3 clinical trials. Read more from ALS News.
