The FNIH Comments on FDA Approval of New Gene Therapies to Treat Sickle Cell Disease

North Bethesda, MD, December 8, 2023—The Foundation for the National Institutes of Health (FNIH) applauds the FDA’s approval of two new gene therapies—Casgevy and Lyfgenia—as treatment options for adolescents and adults with sickle cell disease (SCD). The FNIH has supported, and continues to support, the underlying gene-editing research at NIH’s National Heart, Lung, and Blood Institute that helped lay the foundation for this breakthrough in a disease that has long been overlooked in the medical community.

“This breakthrough brings real hope to people whose lives are challenged by the complications of sickle cell disease,” said Julie Gerberding, President and CEO of the FNIH. “The news couldn’t have come soon enough for those who may endure years of chronic pain, organ failure, and shortened life expectancy.”

People with SCD have abnormally shaped red blood cells that can block the flow of blood throughout the body, leading to recurring pain, multi-organ damage, strokes, and early mortality. 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 is working to advance the next phase of sickle cell treatment by investing in research to make SCD gene therapy more available worldwide. Accessibility is especially important for people in low- and middle-income countries that may not have a cell-processing center and related resources necessary to procure cells from a patient, edit the defective genes outside the body, and then transplant the gene-edited cells back into the patient’s blood. The FNIH and NIH’s National Heart, Lung, and Blood Institute launched a project earlier this year to explore techniques for editing genes entirely inside a patient’s body, eliminating the need for an advanced healthcare infrastructure.

“While approval of these gene therapies will be life-changing news for some patients, we need to focus on ensuring all patients can benefit,” said Michael Santos, Senior Vice President and Chief Population Health Science Officer at the FNIH. “The FNIH is supporting research at the National Heart, Lung, and Blood Institute to enable the gene editing to happen entirely inside the body, which 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.”