The disclosure provides chemical compounds useful as activators of PKR. PKR activating compounds are useful in the treatment of disease and disorders associated with modulation of PKR and/or PKM2, such as pyruvate kinase deficiency (PKD).
The resolution of racemic α-, β-, and γ-hydroxy-ω-alkenylphosphonates was achieved by enzymatic hydrolysis of the corresponding acetates. The optically active alcohols were transformed into β-hydroxyaldehydes and allowed to react with dihydroxyacetone phosphate (DHAP) via enzymatic aldol addition catalyzed by rabbit muscle fructose 1,6-bisphosphate aldolase (FruArab). After enzymatic dephosphorylation
Compositions for the activation of PKR are provided, as well as therapeutic administration of the compositions for the treatment of pyruvate kinase-related medical conditions, such as pyruvate kinase deficiency (PKD).
本研究提供了用于激活 PKR 的组合物,以及用于治疗丙酮酸激酶相关疾病(如丙酮酸激酶缺乏症 (PKD))的组合物的治疗用药。
Activating pyruvate kinase R and mutants thereof
申请人:FORMA Therapeutics, Inc.
公开号:US11001588B2
公开(公告)日:2021-05-11
The disclosure provides chemical compounds useful as activators of PKR. PKR activating compounds are useful in the treatment of disease and disorders associated with modulation of PKR and/or PKM2, such as pyruvate kinase deficiency (PKD).
本公开提供了可用作 PKR 激活剂的化合物。PKR激活化合物可用于治疗与PKR和/或PKM2调节有关的疾病和失调,如丙酮酸激酶缺乏症(PKD)。
Pyrrolopyrrole compositions as pyruvate kinase (PKR) activators
申请人:FORMA Therapeutics, Inc.
公开号:US11014927B2
公开(公告)日:2021-05-25
The disclosure relates to modulating pyruvate kinase and provides novel chemical compounds of formula (I) useful as activators of PKR, as well as various uses of these compounds. PKR activating compounds are useful in the treatment of diseases and disorders associated with PKR and/or PKM2, such as pyruvate kinase deficiency (PKD), sickle cell disease (SCD), and thalassemia.
本公开涉及调节丙酮酸激酶,并提供了可用作 PKR 激活剂的式 (I) 新型化合物,以及这些化合物的各种用途。PKR激活化合物可用于治疗与PKR和/或PKM2相关的疾病和失调,如丙酮酸激酶缺乏症(PKD)、镰状细胞病(SCD)和地中海贫血症。