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The first two patients to receive a CRISPR-based treatment for the inherited blood disorders sickle cell disease and beta thalassemia have benefited from the experimental therapy and experienced only temporary and treatable side effects, the companies developing the treatment announced on Tuesday.

The two patients, enrolled in a pair of ongoing clinical trials, have been free from blood transfusions and disease symptoms for a relatively short time, but the encouraging data offer hope that genome editing might one day offer a safe, durable cure for both blood diseases.


The companies, CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals, have competition. Last June, a one-time gene therapy for beta thalassemia from Bluebird Bio secured approval in Europe, with a U.S. approval expected next year. Several other companies and academic scientists are also pursuing curative treatments for beta thalassemia and sickle cell disease based on genome editing or gene therapy.

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  • The results confirm the usefulness of gene editing in treating incurable genetic diseases. These patients also offer a unique opportunity to evaluate possible unwanted genetic damages; a proposition that will take time since PCR seems that does not work well in the case of CRISPR. Finally, it will be interesting to have an idea of what will be the cost of these treatments, if possible comparing it with the economic burden of an untreated disease. A cost that will be important to assess the economic impact of these therapies on health care.

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