Long-Term Dosing in Sickle Cell Disease Subjects with GBT440, a Novel HbS Polymerization Inhibitor

• Published in: Blood Vol. 128; no. 22; p. 2488
• Main Authors: Lehrer-Graiwer, Josh, Howard, Jo, Hemmaway, Claire Jane, Awogbade, Moji, Telfer, Paul, Layton, Mark, Porter, John B., Mant, Timothy, Dufu, Kobina, Hutchaleelaha, Athiwat, Oksenberg, Donna, Patel, Mira, Tonda, Margaret Eleanor, Bridges, Kenneth, Ramos, Eleanor
• Format: Journal Article
• Language: English
• Published: Elsevier Inc 02.12.2016

Background: Sickle cell disease (SCD) is caused by polymerization of Hemoglobin S (HbS), resulting in hemolysis and vaso-occlusion. No therapy achieving pancellular, direct inhibition of HbS polymerization is available. GBT440 is a novel small molecule which increases hemoglobin oxygen affinity and...