Modulating hemoglobin allostery for treatment of sickle cell disease: current progress and intellectual property.

ABSTRACT:

Introduction

Sickle cell disease (SCD) is a debilitating inherited disorder that affects millions worldwide. Four novel SCD therapeutics have been approved, including the hemoglobin (Hb) modulator Voxelotor.

Areas covered

This review provides an overview of discovery efforts toward modulating Hb allosteric behavior as a treatment for SCD, with a focus on aromatic aldehydes that increase Hb oxygen affinity to prevent the primary pathophysiology of hypoxia-induce erythrocyte sickling.

Expert opinion

The quest to develop small molecules, especially aromatic aldehydes, to modulate Hb allosteric properties for SCD began in the 1970s; however, early promise was dogged by concerns that stalled support for research efforts. Persistent efforts eventually culminated in the disc

SUBMITTER: Pagare PP 

PROVIDER: S-EPMC8881396 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature