PF-07059013：一种非共价血红蛋白调节剂对镰状细胞病小鼠模型的疾病状态有积极影响。 - Suppr

PF-07059013: A non-covalent hemoglobin modulator favorably impacts disease state in a mouse model of sickle cell disease.

作者信息

Knee Kelly M, Jasuja Reema, Barakat Amey, Rao Dharani, Wenzel Zane, Jasti Jayasankar, Novak Jonathan, Beaumont Kevin, Piotrowski David W, Jeffrey Phil, Bulawa Christine, Murphy John E, Janz Jay M

机构信息

Rare Disease Research Unit, Pfizer Worldwide Research, Development, and Medical, Cambridge, Massachusetts, USA.

Primary Pharmacology Group, Pfizer Medicine Design, Pfizer Worldwide Research, Development and Medical, Groton, Connecticut, USA.

出版信息

Am J Hematol. 2021 Aug 1;96(8):E272-E275. doi: 10.1002/ajh.26204. Epub 2021 May 13.

DOI:10.1002/ajh.26204

PMID:33891711

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8360071/

Abstract

摘要

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bc95/8360071/2132e5c3275a/AJH-96-E272-g001.jpg

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PF-07059013: A non-covalent hemoglobin modulator favorably impacts disease state in a mouse model of sickle cell disease.PF-07059013：一种非共价血红蛋白调节剂对镰状细胞病小鼠模型的疾病状态有积极影响。

Am J Hematol. 2021 Aug 1;96(8):E272-E275. doi: 10.1002/ajh.26204. Epub 2021 May 13.

Studies on abnormal hemoglobins. III. The interrelationship of type S (sickle cell) hemoglobin and type F (alkali resistant) hemoglobin in sickle cell anemia.异常血红蛋白的研究。III. 镰状细胞贫血中S型（镰状细胞）血红蛋白与F型（耐碱）血红蛋白的相互关系。

Blood. 1952 Jan;7(1):47-52.

Hemoglobin aggregation in single red blood cells of sickle cell anemia.镰状细胞贫血单个红细胞中的血红蛋白聚集

Science. 1983 Jun 10;220(4602):1173-5. doi: 10.1126/science.6857241.

Genetic model for observed distributions of proportions of haemoglobin in sickle-cell trait.镰状细胞性状中血红蛋白比例观察分布的遗传模型。

Nature. 1977 Aug 18;268(5621):635-6. doi: 10.1038/268635a0.

Studies on abnormal hemoglobins. V. The distribution of type S, sickle cell, hemoglobin and type F, alkali resistant, hemoglobin within the red cell population in sickle cell anemia.异常血红蛋白的研究。V. 镰状细胞贫血患者红细胞群体中S型（镰状细胞血红蛋白）和F型（抗碱血红蛋白）血红蛋白的分布。

Blood. 1952 Dec;7(12):1216-26.

Studies on abnormal hemoglobins. VI. Electrophoretic demonstration of type S (sickle cell) hemoglobin in erythrocytes incapable of showing the sickle cell phenomenon.异常血红蛋白的研究。VI. 无法表现出镰状细胞现象的红细胞中S型（镰状细胞）血红蛋白的电泳显示

Blood. 1953 Mar;8(3):270-5.

An extracorporeal system that modifies hemoglobin in patients with sickle cell anemia.一种用于改变镰状细胞贫血患者血红蛋白的体外系统。

Artif Organs. 1979 Feb;3(1):62-72. doi: 10.1111/j.1525-1594.1979.tb03806.x.

Knockout-transgenic mouse model of sickle cell disease.镰状细胞病的基因敲除转基因小鼠模型

Science. 1997 Oct 31;278(5339):873-6. doi: 10.1126/science.278.5339.873.

Further studies on hemoglobin C. II. The hematologic effects of hemoglobin C alone and in combination with sickle cell hemoglobin.血红蛋白C的进一步研究。II. 单独的血红蛋白C以及与镰状细胞血红蛋白联合时的血液学效应。

Blood. 1953 Aug;8(8):735-46.

Further studies on hemoglobin C. I. A description of three additional families segregating for hemoglobin C and sickle cell hemoglobin.血红蛋白C的进一步研究。I. 对另外三个分离出血红蛋白C和镰状细胞血红蛋白的家族的描述。

Blood. 1953 Aug;8(8):724-34.

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A Pharmacometrics Model to Characterize a New Type of Target-Mediated Drug Disposition (TMDD) - Nonlinear Pharmacokinetics of Small-Molecule PF-07059013 Mediated By Its High-capacity Pharmacological Target Hemoglobin With Positive Cooperative Binding.一种药代动力学模型来描述一种新型的靶向介导的药物处置（TMDD） - 小分子 PF-07059013 与高容量药理学靶标血红蛋白的非线性药代动力学，血红蛋白与正协同结合。

AAPS J. 2023 Apr 13;25(3):41. doi: 10.1208/s12248-023-00808-3.

Fluorescence Lifetime Measurement of Prefibrillar Sickle Hemoglobin Oligomers as a Platform for Drug Discovery in Sickle Cell Disease.作为镰状细胞病药物发现平台的预纤维状镰状血红蛋白寡聚物的荧光寿命测量。

Biomacromolecules. 2022 Sep 12;23(9):3822-3830. doi: 10.1021/acs.biomac.2c00671. Epub 2022 Aug 9.

本文引用的文献

PF-07059013: A Noncovalent Modulator of Hemoglobin for Treatment of Sickle Cell Disease.PF-07059013：一种非共价调节血红蛋白的药物，用于治疗镰状细胞病。

J Med Chem. 2021 Jan 14;64(1):326-342. doi: 10.1021/acs.jmedchem.0c01518. Epub 2020 Dec 24.

A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.一项针对镰状细胞病患者的 voxotor 的 3 期随机试验。

N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14.

GBT440 increases haemoglobin oxygen affinity, reduces sickling and prolongs RBC half-life in a murine model of sickle cell disease.在镰状细胞病小鼠模型中，GBT440可提高血红蛋白对氧的亲和力，减少镰变并延长红细胞半衰期。

Br J Haematol. 2016 Oct;175(1):141-53. doi: 10.1111/bjh.14214. Epub 2016 Jul 5.

Sickle-cell disease.镰状细胞病。

Lancet. 2010 Dec 11;376(9757):2018-31. doi: 10.1016/S0140-6736(10)61029-X. Epub 2010 Dec 3.

Global epidemiology of haemoglobin disorders and derived service indicators.血红蛋白疾病的全球流行病学及相关服务指标

Bull World Health Organ. 2008 Jun;86(6):480-7. doi: 10.2471/blt.06.036673.

Red cell exchange in sickle cell disease.镰状细胞病中的红细胞置换

Hematology Am Soc Hematol Educ Program. 2006:48-53. doi: 10.1182/asheducation-2006.1.48.

Deconstructing sickle cell disease: reappraisal of the role of hemolysis in the development of clinical subphenotypes.解析镰状细胞病：重新评估溶血在临床亚表型发展中的作用。

Blood Rev. 2007 Jan;21(1):37-47. doi: 10.1016/j.blre.2006.07.001. Epub 2006 Nov 7.

Knockout-transgenic mouse model of sickle cell disease.镰状细胞病的基因敲除转基因小鼠模型

Science. 1997 Oct 31;278(5339):873-6. doi: 10.1126/science.278.5339.873.

Potassium cyanate as an inhibitor of the sickling of erythrocytes in vitro.氰酸钾作为体外红细胞镰变的抑制剂。

Proc Natl Acad Sci U S A. 1971 Jun;68(6):1180-3. doi: 10.1073/pnas.68.6.1180.

Increased survival of sickle-cell erythrocytes after treatment in vitro with sodium cyanate.用氰酸钠体外处理后镰状细胞红细胞的存活率增加。

Proc Natl Acad Sci U S A. 1971 Nov;68(11):2791-3. doi: 10.1073/pnas.68.11.2791.

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PF-07059013: A non-covalent hemoglobin modulator favorably impacts disease state in a mouse model of sickle cell disease.

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