Abstract

Last month, the health care giant Baxter agreed to buy AesRx, a virtual biotech firm developing a drug for sickle cell disease. If successful, the drug, Aes-103, could be the first dedicated treatment for the more than 85,000 people in the U.S. with sickle cell. But despite a sizable market and significant need, the drug might just as easily have disappeared into obscurity. Developing drugs for sickle cell has proven daunting, and investors were not interested in Aes-103. The compound was kept alive in large part because of a National Institutes of Health program called Therapeutics for Rare & Neglected Diseases (TRND). It was started in 2009 to forge partnerships with companies or institutions in need of help with drug development projects that are high risk but potentially offer high therapeutic reward. Sickle cell is the result of a mutation in hemoglobin, the component of red blood cells tasked with ...

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