A drug candidate developed by researchers at the NIH's National Center for Advancing Translational Sciences (NCATS) and its collaborators to treat sickle cell disease has been acquired by Baxter International's BioScience business. The drug candidate, Aes-103, is the first specifically developed to target the underlying molecular mechanism of sickle cell disease. Baxter now will advance the clinical development activities required for regulatory approval and commercialization.
Sickle cell disease is a genetic blood disorder that affects millions worldwide, including approximately 100,000 people in the United States among them, 1 in 500 African-Americans.
This is the first time a company has acquired a drug candidate developed with NCATS' Therapeutics for Rare and Neglected Diseases (TRND) program resources. Baxter International recently acquired AesRx, LLC, Newton, Massachusetts the TRND program collaborator including Aes-103. TRND and AesRx researchers worked together to develop Aes-103 through a Phase II clinical trial to evaluate safety and effectiveness. The trial data indicated that Aes-103 significantly reduced patients' pain.
"This is a wonderful example of why NCATS was created," said NIH Director Francis S. Collins, M.D., Ph.D. "The progress made thus far in the development of Aes-103 demonstrates NCATS' catalytic role in bringing together the necessary players, whether academic, nonprofit or industry, to overcome obstacles to translation and advance badly needed treatments to patients."
Individuals living with sickle cell disease have defective hemoglobin, the protein in red blood cells that carries oxygen. This defect causes their cells to become rigid and crescent-shaped, blocking small blood vessels and causing inflammation, pain and strokes, and decreased blood flow.
Aes-103 works by binding directly to hemoglobin and changing its structure, thereby reducing the sickling of red blood cells. This st
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NIH/National Center for Advancing Translational Sciences (NCATS)