Voxelotor, also known as GBT-440, is a hemoglobin S allosteric modulator. GBT440 Inhibits Sickling of Sickle Cell Trait Blood Under In Vitro Conditions Mimicking Strenuous Exercise. GBT440 increases haemoglobin oxygen affinity, reduces sickling and prolongs RBC half-life in a murine model of sickle cell disease. GBT440 increases haemoglobin oxygen affinity, reduces sickling and prolongs RBC half-life in a murine model of sickle cell disease.
Metcalf, Brian; Chuang, Chihyuan; Dufu, Kobina; Patel, Mira P.; Silva-Garcia, Abel; Johnson, Carl; Lu, Qing; Partridge, James R.; Patskovska, Larysa; Patskovsky, Yury; Almo, Steven C.; Jacobson, Matthew P.; Hua, Lan; Xu, Qing; Gwaltney, Stephen L.; Yee, Calvin; Harris, Jason, Morgan, Bradley P.; James, Joyce; Xu, Donghong; Hutchaleelaha, Athiwat; Paulvannan, Kumar; Oksenberg, Donna; Li, Zhe. Discovery of GBT440, an Orally Bioavailable R-State Stabilizer of Sickle Cell Hemoglobin. ACS Medicinal Chemistry Letters. Volume 8. Issue 3. Pages 321-326. Journal; Online Computer File. (2017).
Li, Zhe; Guz, Nathan; Shao, Yiyang; Cocuz, Julieana; Frieser, Markus; Yiannikouros, George Petros; Liao, Liang. Process for synthesizing 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)-pyridin-3-yl)methoxy)benzaldehyde. Assignee Global Blood Therapeutics, Inc., USA. WO 2017197083. (2017).