FDA clears first CRISPR treatment for a second disease, beta thalassemia | CNN
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The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease.

The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease.

The groundbreaking treatment can now also be used to treat transfusion-dependent beta thalassemia in people 12 and older. Like sickle cell, beta thalassemia is an inherited blood disorder.

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To make Casgevy, a person’s stem cells are genetically modified using a precision gene editing technique called CRISPR/Cas9. The modified cells are then transplanted back into the body, where they grow and multiply and increase the production of hemoglobin, which decreases symptoms.

The treatment lists for $2.2 million for both sickle cell disease and beta thalassemia.

  • @LufyCZEnglish
    4•1 year ago

    Developing, testing, trialing and pushing drugs through the FDA process is very expensive.

    On the other side, not that many people have the disease, which means there won’t be that much demand for it.

    Would you rather it not have been developed at all? Because it wouldn’t be if there was no incentive for it.

    All that said though, the treatment should be paid for with public money, like in the rest of the world.

    • @oDDmONEnglish
      1•1 year ago

      Sadly, many don’t know that it was public money in the first place that developed, and continues to develop, treatments and therapeutics which companies then acquire, tweak and charge as if they did the R&D themselves.

      Then there’s M&A as replacement for R&D, which again benefits patients 0 & shareholders bazillions.

      • @MarcbmannEnglish
        1•1 year ago

        Got any sources for this being funded by public money? Would like to see more about that

        • @oDDmONEnglish
          1•1 year ago

          Sure thing! Here’s one:

          ”The discovery stage of CRISPR that goes from its study as a biological phenomenon to its conception as a mechanism that can be used as a genomic editing tool took place mainly in American universities with public funding from federal agencies such as the Department of Energy and the National Science Foundation”.

          https://communities.springernature.com/posts/funding-a-technological-revolution-who-pays-for-what-crispr-research

          In other words, your tax dollars at work.

          • @oDDmONEnglish
            1•1 year ago

            And another:

            Since the Second World War, in the period known as the golden age of capitalism, government agencies have been the main source of funding for scientific research in the U.S. academy (Geiger, 2008). Breakthrough technologies have emerged from long-term investments in R&D under the banner of a “public good” mission (Mazzucato, 2015). Amid the growing influence of productivist coalitions since the 1930s—farm commodity groups, land-grant administrators, agribusiness firms, and federal agricultural agencies (Buttel, 2005)—government agencies have largely taken the lead in actively shaping markets and shouldering the risk of early-stage transformative research investments (Mazzucato, 2015). Private-sector actors typically limited their role to lower risk forms of technology integration, development, and marketing later in the innovation process.

            https://direct.mit.edu/qss/article/3/2/443/110371/Funding-CRISPR-Understanding-the-role-of