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Groundbreaking DDF-funded Sickle Cell Cure Moves Closer to FDA Approval

The New York Times published an article about a significant breakthrough in sickle cell disease treatment, as an advisory committee has confirmed the safety and efficacy of an innovative cure for patients. If approved by the U.S. Food and Drug Administration (FDA), it could become the first curative therapy to treat a genetic disease using CRISPR gene-editing technology. The treatment, known as exa-cel and developed jointly by Vertex Pharmaceuticals and CRISPR Therapeutics, stands as a potential cure for a disease affecting over 100,000 people in the United States, many of whom are of sub-Saharan African descent. Two of the scientists behind this work, Daniel E. Bauer, M.D., Ph.D., and Stuart H. Orkin, M.D., together received Doris Duke Foundation grants in 2013 and 2015 to identify core genetic sequences that upon CRISPR intervention created healthy red blood cells.

Read more in The New York Times.