The approval of Casgevy and Lyfgenia in 2023 marks a pivotal moment in the treatment of sickle cell disease, a genetic disorder affecting around 100,000 people in the U.S., predominantly among non-Hispanic Black or African American populations. These therapies offer the possibility of a cure, with studies showing that 88% to 94% of patients experience complete freedom from severe pain crises.
Despite this, the uptake of these therapies has been slow, with only 64 patients treated with Casgevy and 115 with Lyfgenia reported by late 2025. The complexity of the treatment process, which includes stem cell collection and conditioning that may lead to infertility, alongside the high costs—ranging from $2.2 million to $3.1 million per treatment—pose significant barriers to access.
Many patients, particularly those reliant on Medicaid, may find it difficult to navigate the healthcare landscape to obtain these potentially life-changing therapies. Experts emphasize the need to address these access issues to ensure that the benefits of these advancements reach all eligible patients