AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency-Reference-Cited by-同舟云学术

AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency

Published:2017-09-14 Issue:11 Volume:130 Page:1347-1356
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Kung Charles¹,Hixon Jeff¹,Kosinski Penelope A.¹,Cianchetta Giovanni¹,Histen Gavin¹,Chen Yue¹,Hill Collin¹,Gross Stefan¹,Si Yaguang¹,Johnson Kendall¹,DeLaBarre Byron¹,Luo Zhiyong²,Gu Zhiwei²,Yao Gui²,Tang Huachun²,Fang Cheng²,Xu Yingxia²,Lv Xiaobing²,Biller Scott¹,Su Shin-San Michael¹,Yang Hua¹,Popovici-Muller Janeta¹,Salituro Francesco¹,Silverman Lee¹,Dang Lenny¹

Affiliation:

1. Agios Pharmaceuticals, Inc., Cambridge, MA; and

2. Shanghai ChemPartner Co., Ltd., Shanghai, China

Abstract

Key Points AG-348 is a small-molecule allosteric activator of WT red cell pyruvate kinase as well as mutant enzymes associated with hemolytic anemia. Activity in vitro, in mice, and in red blood cells suggests it may address the underlying molecular pathology in PK deficiency patients.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/130/11/1347/1402486/blood753525.pdf

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