Stuart Orkin and Daniel Bauer receive funding for sickle cell disease research from the Doris Duke Charitable Foundation
September 12, 2019
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
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.
the Doris Duke Charitable Foundation
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.