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FDA clears first CRISPR treatment for a second disease, beta thalassemia (www.cnn.com)

submitted 11 months ago by MicroWave to c/health

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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 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.

...

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.

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[–] oDDmON 1 points 11 months 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