PF-07059013

Names

[ Name ]:
PF-07059013

Biological Activity

[Description]:

PF-07059013 is an orally active and potent noncovalent modulator of sickled hemoglobin (HbS). PF-07059013 specifically binds to Hb with nanomolar affinity and displays strong partitioning into red blood cells (RBCs). PF-07059013 can be used for sickle cell disease (SCD) research[1][2].

[Related Catalog]:

Research Areas >> Cardiovascular Disease

Signaling Pathways >> Others >> Others

[In Vivo]

PF-07059013 (10-490 mg/kg，口服灌胃，每日2次，连用2周) 显示 Townes SCD 小鼠的镰刀减少[1]。 Animal Model: Townes SCD mice (8-10 weeks old, male)[1] Dosage: 10, 40, 200, and 490 mg/kg Administration: Oral gavage, twice daily, for 2 weeks Result: Showed a 37.8% (±9.0%) reduction in sickling at 200 mg/kg compared to vehicle treated mice.

[References]

[1]. Gopalsamy A, et al. PF-07059013: A Noncovalent Modulator of Hemoglobin for Treatment of Sickle Cell Disease. J Med Chem. 2021 Jan 14;64(1):326-342.

[2]. Aaron Baldwin, et al. Route Optimization of the Non-covalent Modulator of Hemoglobin PF-07059013 for the Treatment of Sickle Cell Disease, Part I: From Discovery Synthesis to First Kilogram-Scale Manufacture. Org. Process Res. Dev. 2023.

Chemical & Physical Properties

[ Molecular Formula ]:
C₁₉H₁₆FN₅O₂

[ Molecular Weight ]:
365.36

Related Compounds

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