Will 50 Americans have been confirmed to have been cured of thalassemia through gene therapy by the end of 2024?
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Bluebird bio recently got FDA approval for their gene therapy to treat patients with beta thalassemia who require regular red blood cell transfusions. They have worked out a payment system with American health care providers, and they are currently setting up treatment centers. It appears that the process has worked in about 40 individuals, but all in overseas clinical trials, with the success rate being 89% for transfusion independence 12 months after the trial.
This market will resolve YES if at least 50 Americans have been confirmed to be transfusion independent for 12 months after receiving gene therapy treatment, from any provider.
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