GBT-440

GBT-440 is a modulator of sickle hemoglobin (HbS) that binds covalently to the N-terminal valine of the alpha chain of HbS. In vitro, GBT-440 increases HbS affinity for oxygen (EC50s = 8.2 and 415 µM for purified HbS and whole blood from sickle cell anemia patients, respectively) and delays oxygen release from and polymerization of HbS in a concentration-dependent manner. GBT-440 (100-150 mg/kg) increases red blood cell half-life and reduces sickling ex vivo in whole blood isolated from a murine model of sickle cell anemia. It also reduces ex vivo sickling of sickle cell trait red blood cells under conditions of hypoxia, acidosis, and dehydration.Formal Name: 2-hydroxy-6-[[2-[1-(1-methylethyl)-1H-pyrazol-5-yl]-3-pyridinyl]methoxy]-benzaldehyde. CAS Number: 1446321-46-5. Synonyms: Voxelotor. Molecular Formula: C19H19N3O3. Formula Weight: 337.4. Purity: >98%. Formulation: (Request formulation change), A crystalline solid. Solubility: DMF: 33 mg/ml, DMSO: 33 mg/ml, DMSO:PBS (pH 7.2) (1:5): 0.16 mg/ml, Ethanol: 20 mg/ml. lambdamax: 272, 276 nm. SMILES: OC1=C(C=O)C(OCC2=CC=CN=C2C3=CC=NN3C(C)C)=CC=C1. InChi Code: InChI=1S/C19H19N3O3/c1-13(2)22-16(8-10-21-22)19-14(5-4-9-20-19)12-25-18-7-3-6-17(24)15(18)11-23/h3-11,13,24H,12H2,1-2H3. InChi Key: FWCVZAQENIZVMY-UHFFFAOYSA-N.