| Name | voxelotor |
| Description | Voxelotor (GBT 440) is a novel hemoglobin S (HbS) polymerization inhibitor primarily used for the treatment of sickle cell disease (SCD) and a cytochrome P450 3A4 inhibitor. Voxelotor targets and covalently binds to the N-terminal valine of the HbS alpha chain, thereby stabilizing sickle cell hemoglobin (HbS). |
| In vitro | METHODS: Voxelotor (3-30 µmol/L) was incubated with hemoglobin (25 µmol/L) in TES or saline buffer at 37 ℃ for 45 min, and changes in the binding affinity of O2 for Hb were measured using a TCS hematology analyzer.
RESULTS: Voxelotor, a small molecule that binds to the N-terminal α-chain of Hb, increases the affinity of HbS for oxygen, delays HbS polymerization in vitro and prevents erythrocyte sickling. [1]
METHODS: HepG2 cells were treated with Voxelotor (1 µg/mL) and their metabolites were analyzed by liquid chromatography, high-resolution MS and tandem MS.
RESULTS: Voxelotor can be used for the identification of phase I and phase II metabolites in vitro. [2] |
| In vivo | METHODS: To assess the activity and efficacy of Voxelotor, Voxelotor (100-150 mg/kg, 0.5% methylcellulose/0.5% SDS) was administered by gavage to a mouse model of SCD twice daily for 9-12 days.
RESULTS: Voxelotor reduced isolated sickle cells and prolonged erythrocyte half-life in the SCD mouse model. [1] |
| Storage | store at low temperature | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
| Solubility Information | DMSO : 125 mg/mL (370.51 mM), Sonication is recommended.
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| Keywords | voxelotor | sickle hemoglobin (HbS) | sickle | SCD | polymerization | Inhibitor | inhibit | HbS | haemoglobin | GBT-440 | GBT440 | disease | cell |
| Related Compound Libraries | FDA-Approved & Pharmacopeia Drug Library | Failed Clinical Trials Compound Library | Bioactive Compound Library | Pediatric Drug Library | ReFRAME Related Library | EMA Approved Drug Library | Drug Repurposing Compound Library | Inhibitor Library | Anti-Cardiovascular Disease Compound Library | NO PAINS Compound Library | FDA-Approved Drug Library | Bioactive Compounds Library Max |
United States
