Sen. Scott Applauds FDA Approval of Two Sickle Cell Disease Gene Therapies

WASHINGTON — Today, U.S. Senator Tim Scott (R-S.C.) celebrated the Food and Drug Administration’s (FDA) decision to approve exa-cel, from CRISPR Therapeutics and Vertex Pharmaceuticals, as well as lovo-cel, from bluebird bio. Both gene therapies are  potential one-time cures for sickle cell disease. Exa-cel now becomes the world’s first CRISPR gene editing therapy designed to treat sickle cell disease.

“Sickle cell disease affects 100,000 Americans, most prominently those in the African American, Latino and other minority communities,” said Senator Scott. “These revolutionary new treatments are medical innovation at its finest and has the potential to significantly improve the lives of and provide hope to those who have been afflicted with this deadly disease.” 

On June 15, 2023, Senator Scott’s Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2023 wasapproved by the Senate Committee on Health, Education, Labor and Pensions (HELP). The legislation reauthorizes the Health Resources and Services Administration’s (HRSA) Sickle Cell Disease Treatment Demonstration Program beyond fiscal year 2023 through fiscal year 2028. This bill helps to make sure that HRSA has the flexibility to provide technical assistance and educational materials more rapidly to grantees and communities in response to emerging subjects, such as gene therapies to treat individuals with sickle cell disease.

Senator Scott’s leadership on Sickle Cell Disease awareness includes:

  • Introducing and signing into law the Sickle Cell and Other Heritable Blood Disorders Research Surveillance, Prevention and Treatment Act in 2018;
  • Creating a bipartisan resolution to dedicate September as Sickle Cell Awareness month; 
  • Questioning the FDA nominee on the future of CRISPR technology in finding a cure for sickle cell disease; and
  • Designating February 28th as Rare Disease Day to promote awareness for all rare diseases, such as sickle cell.