What the latest progress on sickle cell means for Black Americans

Key Points:

• Daniel Cressy is the first person in Louisiana to be functionally cured of sickle cell disease through gene therapy, offering hope to the approximately 100,000 individuals in the U.S. living with the disease.
• The FDA approved two gene therapies in 2023, Casgevy and Lyfgenia, which involve editing or inserting genes into blood stem cells to produce healthy red blood cells, but the treatment is complex, lengthy, and costly—ranging from $2 million to $3 million.
• Access to gene therapy is limited by factors including the scarcity of treatment centers, the physical demands of the procedure, potential side effects like infertility and increased cancer risk, and the inability to reverse existing organ damage in some patients.
• Expanding access requires coordinated efforts from payers, drug manufacturers, policymakers, and the federal government to improve insurance coverage, increase treatment center availability, establish supportive regulations, and fund research.
• Despite current barriers, increasing numbers of patients receiving gene therapy provide hope that sickle cell disease could eventually be functionally cured for many, especially within the Black community disproportionately affected by the condition.