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Health Agencies Update
August 27, 2014

Sickle Cell Drug Candidate Under Development

Author Affiliations

Copyright 2014 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.

JAMA. 2014;312(8):779. doi:10.1001/jama.2014.10040

For the first time, a pharmaceutical company has acquired a drug candidate developed with resources from a National Institutes of Health (NIH) program for rare and neglected diseases (http://1.usa.gov/1kDK7XW).

The drug, called Aes-103, treats sickle cell disease by binding directly to hemoglobin and changing its structure, thereby reducing the sickling of red blood cells. It is the first drug specifically developed to target the underlying molecular mechanism of sickle cell disease.