FDA poised to approve first gene-editing therapy, made by local drug firms - The Boston Globe
www.bostonglobe.com
The life-changing drug, developed by Boston-based Vertex and its Swiss partner CRISPR Therapeutics, targets sickle cell disease, an inherited blood disorder that causes crippling pain.

Marie Tornyenu missed more than 100 days of high school while hospitalized repeatedly for sickle cell disease, an inherited blood disorder that causes crippling pain. Somehow, she still managed to complete at least eight advanced placement courses, play clarinet in the school band, and get admitted to Boston University.

Then in December 2021, after transferring to Cornell University, she received an experimental medicine that used a revolutionary gene-editing technology to treat inherited diseases. It freed her from excruciating bouts of pain and the frequent blood transfusions that are a mainstay of sickle cell treatment.

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The life-changing drug, developed by Boston-based Vertex and its Swiss partner CRISPR Therapeutics, is expected to be approved by the Food and Drug Administration by Friday for people with severe cases of the disease. Called Casgevy, it would usher in a new era not only for those with sickle cell but also for medicine: The drug would be the first gene-editing therapy authorized by US regulators, and uses a tool called CRISPR.

The likely approval — Casgevy was cleared by British regulators last month — raises both the promise of cures for diseases as well as the ethical concerns that come with the power to manipulate the building blocks of human life. With an expected price tag in the seven figures, it also touches on issues of equity in medicine.

  • tacosanonymous
    1·
    1 year ago

    At a cost over one million dollars, I’m not sure it’ll be that helpful to the public.

    • Flying Squid@lemmy.world
      24·
      1 year ago

      Depends on how much insurance is willing to cover of that. Insurance is willing to cover a surprising amount, although you will still probably be stuck with a fairly large bill (but in the thousands, not higher than that). Of course, if you don’t have insurance, you’re definitely fucked.

      • LastYearsPumpkin@feddit.chEnglish
        2·
        1 year ago

        Since this is treating a chronic, lifetime condition, with a single treatment, this is certainly something that you can take some time and pick a better insurance plan for a few years until the treatment is over.

        It sucks that it’s even a consideration, but this isn’t a condition that suddenly comes up. You’ve probably been talking to your doctors about it for decades by the time you get to this point.