Gene editing offers hope for sickle cell disease
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In December 2023, the United States approved Casgevy and Lyfgenia, new gene therapies treating sickle cell disease (SCD), a genetic condition first documented in 1910.
Kevin Davies, PhD, recently published Curved Air, a book detailing the history of SCD and the development of these therapies, inspired by the story of Victoria Gray, the first patient treated with CRISPR cell therapy.
Davies learned of the potential of gene editing to treat SCD while writing a previous book about the CRISPR revolution, noting the irony that this advanced technology addressed a long-neglected disease. The book’s title, Curved Air, references the crescent shape of sickle cells and their reduced ability to carry oxygen.
Davies’ research revealed the first documented U.S. case of SCD occurred in 1904, with a dental student from Grenada, and uncovered evidence of the disease existing in enslaved African Americans in the early 1800s. He also detailed how researchers discovered that manipulating levels of fetal hemoglobin could compensate for the SCD mutation, a finding that will be recognized with a 2026 Breakthrough Prize.
Davies also highlighted inequities in SCD care, sharing the story of Brittany Hightower, a patient who experienced discrimination and was denied care during a pain crisis before her death. While Casgevy and Lyfgenia represent a major advancement, costing over $2 million per treatment, most U.S. insurance companies and many states are covering the cost. Scientists are continuing to work towards more accessible therapies, including small-molecule drugs, to benefit patients worldwide.
