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    FDA approves groundbreaking CRISPR-based gene therapies for sickle cell disease

    The approval, disclosed on Friday morning, includes two milestone treatments named Casgevy and Lyfgenia. These therapies represent the first cell-based gene therapies for sickle cell disease treatment in patients aged 12 years and older.

    Sickle cell disease, affecting around 100,000 people in the U.S., is prevalent among African Americans and constitutes a group of inherited blood disorders. Jalen Matthews, a sickle cell patient, shared her challenging journey, including chronic pain, frequent hospital treatments, a blood clot, and experiencing a stroke at the age of twelve.

    “The clinical trials done were done with sickle cell patients showed an amazing effect,” said Dr. Nicole Verdun, an FDA Super Office Director. “They had a reduction in pain and episodes that lead to severe and debilitating disease.”

    During the two-year follow-up on clinical trial patients, the majority being African American due to the group’s disproportionate impact, doctors observed promising results. Although the therapies show great potential, medical professionals acknowledge that there are still uncertainties.

    Regarding costs, Casgevy is priced at $2.2 million for a one-time treatment, while Lyfgenia costs $3.1 million. Treating everyone with these therapies will amount to billions. However, for those with insurance, coverage is assured. Some health experts argue that the cost is economical compared to the frequent hospitalizations sickle cell patients undergo over their lifetimes. Dr. Verdun emphasized the FDA’s commitment to addressing access issues for all individuals, an aspect they will continue to work on.



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