Sickle cell gene therapies offer new hope, but cost and access block many patients
Two FDA-approved gene therapies can free severe sickle cell patients from pain crises, yet high prices, a year-long treatment process and few specialists keep them out of reach for many.
At a glance
- The FDA approved Casgevy and Lyfgenia in 2023, the first gene therapies for severe sickle cell disease.
- Gene therapy frees 88%-94% of patients from severe pain crises, but only a few hundred have been treated so far.
- List prices of $2.2 million to $3.1 million, complex care and few specialists limit access.
Two gene therapies approved in the United States in 2023 are giving people with severe sickle cell disease a path to what patient advocates describe as a potential cure — but cost, a demanding treatment process and a shortage of specialists are keeping the therapies out of reach for many patients.
The FDA approved Casgevy and Lyfgenia in 2023, the first gene therapies for severe forms of the inherited blood disorder. Dr. Edward Donnell Ivy, chief medical officer of the Sickle Cell Disease Association of America, said the treatments "open up a whole world of individuals potentially being cured."
Sickle cell disease affects around 100,000 people in the U.S., according to the Centers for Disease Control and Prevention, and more than 90% of patients are non-Hispanic Black or African American. The disorder turns normally flexible, round red blood cells rigid and crescent-shaped, blocking blood vessels and reducing oxygen delivery to tissues. Complications include chronic fatigue, anemia, blood clots, stroke and severe "pain" crises that can last from hours to weeks.
Slow progress, then a breakthrough
Progress was long delayed. A 2020 analysis cited in the report found that National Institutes of Health research funding for sickle cell disease averaged $812 per patient from 2008 through 2018, compared with $2,807 per patient for cystic fibrosis, which affects about 40,000 people in the U.S.
Hydroxyurea, approved in 1998, became a cornerstone treatment and has been shown in studies to cut the frequency of pain crises by half. A cure remained elusive until gene therapy: bone marrow transplants were limited to the few patients who could find a donor match.
Gene therapy achieves complete freedom from severe vaso-occlusive crises in 88% to 94% of patients, yet uptake has been slow. Vertex Pharmaceuticals reported that 64 patients worldwide had received Casgevy infusions by the end of 2025, with 147 more beginning cell collection. Genetix Biotherapeutics said 115 U.S. patients had been treated with Lyfgenia since its approval, with another 216 starting cell collection.
Cost and care gaps
List prices range from $2.2 million to $3.1 million for a single course, excluding hospital stays, chemotherapy and specialized care. Treatment involves collecting and modifying a patient's blood-forming stem cells and returning them, after chemotherapy conditioning that can cause permanent infertility; the process can take up to a year.
Medicaid covers the therapy, and the Centers for Medicare and Medicaid Services launched the Cell and Gene Therapy Access Model in 2024 to set outcome-based pricing with states and manufacturers. Thirty-two states, the District of Columbia and Puerto Rico take part. Dr. Alexis Thompson, a physician at Children's Hospital of Philadelphia and spokesperson for the American Society of Hematology, said covered services can vary by state.
Provider shortages may be the bigger obstacle. Dr. Christina Abrams, director of the Pediatric Sickle Center at the Medical University of South Carolina, said some patients drive up to three hours for sickle cell expertise. Studies estimate that 25% to 35% of patients meet the current clinical criteria for gene therapy.
Source: CNBC.
Questions and answers
- How much does sickle cell gene therapy cost?
- The list price for a single-course treatment ranges from $2.2 million to $3.1 million. That figure does not include hospital stays, chemotherapy or the specialized care needed.
- How many people have received sickle cell gene therapy?
- By the end of 2025, 64 patients worldwide had received Casgevy infusions, with 147 more starting cell collection, Vertex reported. Genetix Biotherapeutics said 115 U.S. patients had been treated with Lyfgenia since 2023.
- Who is eligible for sickle cell gene therapy?
- Both therapies are approved for severe forms of the disease. Casgevy can be given from age 2 and Lyfgenia from age 12, and studies estimate 25% to 35% of patients meet current clinical criteria.
