Sickle cell disease disproportionately affects African-Americans and is considered both rare and neglected in the United States. African-Americans with sickle cell often face significant health disparities in clinical care. Life expectancy for people with sickle cell disease is only to mid- to late 40s.
Prior to AesRx's collaboration with TRND researchers, and despite promising data on Aes-103, the company had difficulty securing private financing because potential investors lacked interest in funding an early-stage project that was considered too risky. AesRx did not have the resources to complete preclinical and early clinical development.
Currently, the only drug approved by the U.S. Food and Drug Administration (FDA) to treat sickle cell disease is hydroxyurea, a drug initially developed to treat cancer. However, the clinical utility of hydroxyurea is limited. Many individuals with sickle cell disease either do not respond to the drug, or they may experience undesirable side effects.
"Sickle cell was the first disease to ever have its molecular cause discovered more than 65 years ago and now a potential treatment based on that discovery has at last been developed," said NCATS Director Christopher P. Austin, M.D. "This success validates the NCATS model, which is based on a novel collaborative approach that de-risks intervention development programs to enable private-sector investment. We look forward to applying this model to the thousands of rare diseases that are currently untreatable so that we realize the NCATS mission of getting more treatments to more patients more quickly."
TRND researchers signed a collaborative agreement with AesRx in 2010 and established a project team made up of NCATS and AesRx scientists as well as a leading sickle cell disease clinical researcher at the National Heart, Lung, and Blood Institute (NHLBI). Other key p
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NIH/National Center for Advancing Translational Sciences (NCATS)