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Hepcidin Therapeutics.

Angeliki Katsarou1, Kostas Pantopoulos2

  • 1Lady Davis Institute for Medical Research, Jewish General Hospital, Department of Medicine, McGill University, Montreal, QC H3T 1E2, Canada. ageliki.katsarou@mail.mcgill.ca.

Pharmaceuticals (Basel, Switzerland)
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Summary

Hepcidin regulates iron levels in the body. This review covers hepcidin therapeutics, including agonists and antagonists, for treating iron disorders like hemochromatosis and anemia, with some agents in clinical trials.

Keywords:
anemiaferroportinhemochromatosishepcidiniron metabolism

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Area of Science:

  • Biochemistry
  • Endocrinology
  • Genetics

Background:

  • Hepcidin is a crucial hormone regulating systemic iron homeostasis.
  • Its expression is modulated by iron and inflammatory signals.
  • Genetic defects in hepcidin regulation cause hepcidinopathies, including hereditary hemochromatosis and iron-refractory iron deficiency anemia.

Purpose of the Study:

  • To review the current state of hepcidin therapeutics.
  • To highlight the role of hepcidin dysregulation in various iron disorders.
  • To discuss preclinical and clinical developments in targeting hepcidin for treatment.

Main Methods:

  • Review of preclinical animal models demonstrating therapeutic potential.
  • Identification and characterization of hepcidin agonists and antagonists.
  • Analysis of clinical trial data for hepcidin-modulating agents.

Main Results:

  • Dysregulation of hepcidin is implicated in iron-loading anemias and anemia of inflammation.
  • Preclinical studies show that normalizing hepcidin levels can treat these conditions.
  • Several hepcidin-targeting therapeutics are in clinical development.

Conclusions:

  • Hepcidin therapeutics represent a promising new avenue for treating iron homeostasis disorders.
  • Targeting hepcidin offers potential treatments for hemochromatosis, iron deficiency anemia, and anemia of inflammation.
  • Ongoing clinical trials will determine the efficacy and safety of these novel therapies.