Boston Children’s Hospital will receive a $1.5 million grant from the Bill & Melinda Gates Foundation to develop more efficient gene therapy treatments for sickle cell disease, as well as methods to enable gene therapy to be used in developing regions of the world, where there are high rates of sickle cell disease.
Sickle cell disease is a major public health concern in the developing world, leading to death or life-long morbidities. An estimated 275,000 infants worldwide are born annually with sickle cell disease, with more than half of those in developing countries dying in early childhood.
Dana-Farber/Boston Children’s is currently running a clinical gene therapy trial for sickle cell disease which suppresses a gene called BCL11A, enabling patients to make a fetal, non-sickling form of hemoglobin.
The gene therapy in this trial is ex vivo, a process that is costly, time intensive, and involves multiple complicated manufacturing steps, so it can’t be easily recreated in the developing world. Using this grant, David Williams, Leland Fikes Professor of Pediatrics at Harvard Medical School, chief scientific officer and senior vice president of Boston Children’s Hospital, and president of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, along with his colleagues, will conduct research to try to develop in vivo methods that can overcome the current bioengineering and manufacturing constraints.
“Ultimately, an in vivo approach, in which a gene or inhibitory RNA is delivered directly to the body, is likely to be optimal for broadening global access to gene therapy for sickle cell disease,” Williams said.
For this collaborative project Williams will be joined by Paula Hammond of the Koch Institute, Christian Brendel of Dana-Farber/Boston Children’s, Harvey Lodish of the Whitehead Institute, and David Scadden at Massachusetts General Hospital.