[Skip to Content]
Access to paid content on this site is currently suspended due to excessive activity being detected from your IP address Please contact the publisher to request reinstatement.
[Skip to Content Landing]
Views 697
Citations 0
Health Agencies Update
August 27, 2014

Sickle Cell Drug Candidate Under Development

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.

First Page Preview View Large
First page PDF preview
First page PDF preview