Expanding Access to Gene Therapy for Sickle Cell Disease Worldwide

Sickle cell disease (SCD) affects an estimated 100,000 people living in the United States and millions more throughout the world, disproportionately affecting populations of African descent.
The FNIH applauds the recent FDA approval of two treatments that use gene-editing technologies to mitigate the inherited gene mutation that causes SCD. These therapies involve procuring a patient’s own bone marrow cells to repair the defective gene outside the body (ex vivo) and then transplanting the modified cells back into the patient’s blood. The FNIH has supported, and continues to support, the underlying gene-editing research at NIH’s National Heart, Lung, and Blood Institute (NHLBI) that helped lay the foundation for this breakthrough.
Still, access to these therapies remains limited for people in low- and middle-income countries that may not have a cell-processing center and related resources necessary for ex vivo gene therapy. This is where the FNIH is seeking to broaden access to critical gene editing therapies for patients with SCD.
Pathway to Impact:
- In partnership with the NHLBI, the FNIH launched a project to explore techniques for administering gene therapy strategies entirely inside a patient’s body (in vivo), which would eliminate the need for an advanced healthcare infrastructure and make SCD treatment more broadly available.
“The FNIH is supporting research that promises to make a one-time cure available to patients in the United States and globally who can’t access the intensive inpatient procedures required currently.”
Michael Santos, PhD, FNIH Senior Vice President and Chief Population Health Science Officer