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The Doris Duke Charitable Foundation this week announced the six projects receiving approximately $5 million through the second Sickle Cell Disease/Advancing Cures grant competition. Dana-Farber/Boston Children’s Cancer and Blood Disorders Center’s Stuart H. Orkin, MD, and Daniel E. Bauer, MD, PhD, each lead one of those projects.
Bauer’s project, titled “Enhanced nuclease delivery for therapeutic gene editing of hematopoietic stem cells in sickle cell disease,” will study aspects of gene editing that could improve its successful clinical application in patients with sickle cell disease, while Orkin’s project, “Structure-based small molecules for HbF reactivation,” investigates new drug-like molecules and potential new drug targets to promote expression of functional hemoglobin. Adult hemoglobin is a protein in red blood cells that is mutated in sickle cell disease, which disrupts its ability to transport oxygen through the body.
“With the rise of CRISPR gene editing and many other promising developments, this is an exciting time for sickle cell disease research and an opportunity to make important strides toward delivering a cure,” said Betsy Myers, Program Director for Medical Research at the Doris Duke Charitable Foundation. “We are thrilled to support these clinical researchers as they harness the power of recent progress in the field in efforts to develop new ways to advance gene therapies and restore red blood cell function.”
This announcement coincides with National Sickle Cell Awareness Month, which began in 1983 to foster public awareness about the genetic disease that researchers estimate affects between 90,000 to 100,000 Americans. It is the most common inherited blood disorder in the United States, and approximately 300,000 people worldwide are born with sickle cell disease each year. Patients with sickle cell disease carry dysfunctional red blood cells that alter regular blood flow, which translates into pain, poor organ oxygenation and organ damage, and a life span of only about 40 years.
The foundation has supported sickle cell disease research through a variety of grant-making mechanisms, including the Innovations in Clinical Research Award (ICRA), which began in 2009 and has helped enrich the field with projects on disease biology, management, and treatment. The Sickle Cell Disease/Advancing Cures awards program was first launched in 2017 to build upon years of learning from these previously funded projects and seek to capitalize on discoveries that allow for further investment in approaches that specifically target sickle cell disease’s underlying causes. The second peer reviewed competition of the Sickle Cell Disease/Advancing Cures awards is meant to further build upon this investment by supporting research that explores aspects of gene modification and drug discovery that could accelerate cures.
Doris Duke, who endowed the foundation and for whom it is named, had a particular personal interest in supporting sickle cell disease research. She articulated this desire in her will, which in part guides the foundation’s funding priorities.
About the Doris Duke Charitable Foundation
The mission of the Doris Duke Charitable Foundation is to improve the quality of people’s lives through grants supporting the performing arts, environmental conservation, child well-being and medical research, and through preservation of the cultural and environmental legacy of Doris Duke’s properties. The foundation’s Medical Research Program supports clinical research that advances the translation of biomedical discoveries into new preventions, diagnoses, and treatments for human diseases. To learn more about the program, visit www.ddcf.org.