FDA poised to approve first gene-editing therapy, made by local drug firms - The Boston Globe
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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.

    • Flying Squid@lemmy.world
      22·
      10 months ago

      I doubt it would help for chronic pain in general. I have atypical trigeminal neuralgia, a rare nerve disorder. After years of trials with various drugs, a combination of Lamictal, Cymbalta, Lyrica and, for a while Rexulti, seemed to work, but I stopped taking Rexulti when I got a new neurologist who wouldn’t prescribe it and I seem to be doing okay without it.

      • cheese_greater@lemmy.world
        2·
        10 months ago

        Pregabalin is such an incredible medication/substance, particularly for neuralgiac conditions. It never ceases to amaze me

        Edit: this just inspired my next AskLemmy.ml series, spank-you all ;)

        • Flying Squid@lemmy.world
          12·
          10 months ago

          It’s certainly done wonders for me. It didn’t help that I had two really bad neurologists at the start when my symptoms first showed up. The first one told me to take Gabapentin whenever I needed it, no limit. I took a huge amount and it didn’t help at all. The second one tried like 10 opioids on me and none of them helped. I didn’t get truly addicted thankfully, but I did have a pretty awful withdrawal which lasted a couple of days. Finally I found the one who suggested Pregabalin.