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sector_ico_Health_trans Human Health

IND Enabling Studies for Antisense Oligonucleotides Targeting Key Regulators of Iron Metabolism, Hepcidin and Hemojuvelin

  • Project Leaders: Paul Goldberg
  • Institutions: Xenon Pharmaceuticals Inc
  • Budget: $7469280
  • Program/Competition: Applied Genomics Programs
  • Genome Centre(s): Genome British Columbia
  • Status: Closed

Iron is essential to bind oxygen and transport electrons for all living organisms, but iron overload and maldistribution have been implicated in the etiology of many disorders, including anemia of chronic disease (ACD).  ACD is the second most common form of anemia worldwide and a commonly acquired disorder that is associated with a variety of conditions including kidney disease, malignancy and other clinical settings of chronic inflammation such as inflammatory bowel disease. Utilizing its genetics platform, Xenon’s discovery of the genes underlying the rare genetic disorder juvenile hemochromatosis led to its selection of hepcidin, a protein produced in the liver, as a drug target to treat patients with ACD. These projects, based at Xenon Pharmaceuticals, comprised preclinical studies to evaluate the toxicology and efficacy of new therapies for iron overload disorders and ACD, and ultimately supported a decision not to progress into further clinical development.