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Tag Archives: access to genome therapy

Gene-based therapies: opportunity vs access

The advent of CAR-T and CRISPR technologies are set to revolutionize cancer and genetic disease therapies, respectively. But with great hope comes great costs. “The Centers for Disease Control and Prevention estimates that approximately 100,000 people in the US have sickle cell disease, making it the most prevalent inherited blood disorder. Remarkably, the new gene therapies offer very promising early efficacy: exa-cel and lovo-cel demonstrated 93.5%...

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