Today is World Sickle Cell Day, an opportunity to recognize the patients, families and communities affected by sickle cell disease — and to underscore the biopharmaceutical industry's ongoing efforts to advancing new treatments for this life-altering condition. 

Background: Sickle cell disease (SCD) is an inherited rare blood disorder caused by a mutation in the gene that tells the body how to make hemoglobin, which is the protein in red blood cells that carries oxygen.

People living with SCD can experience chronic, severe and unpredictable pain, resulting in frequent, expensive hospital visits, limiting patients' ability to participate in everyday activities. The disease typically manifests in children before they turn one, with life expectancy averaging just 54 years, about 20 years shorter than average U.S. life expectancy.

Who is affected: SCD affects approximately 100,000 people in the United States and disproportionately impacts people of Black or African-American descent, who make up more than 90% of the SCD population in the United States.

Changing the game for patients: For decades, treatment options for SCD primarily helped to manage symptoms. That all changed in 2023 when the FDA approved two cell-based gene therapies to treat SCD — marking a major breakthrough and offering new hope to patients across the country to alter the trajectory of this disease and its debilitating consequences. 

Why it matters: Gene therapies target SCD’s genetic cause and offer long-term, or even curative benefits with a single treatment. These advances could also help patients build a better future, including in the workplace:

• More than half of patients with SCD report that chronic pain and other disease burdens affect their employment status, forcing some to cut back their hours or leave the workforce altogether.
• By reducing painful complications from SCD, gene therapy can help patients avoid disruptions that could affect their ability to work and maintain employment.