FRANKFURT, Germany — Updated results released Friday highlighted the lasting benefits of a CRISPR-based medicine for blood disorders developed by Vertex Pharmaceuticals and CRISPR Therapeutics, as the treatment awaits regulatory decisions in the United States and Europe.
The treatment, a one-time infusion known as exa-cel, uses CRISPR to fix the genetic faults at the root of sickle cell disease and beta thalassemia. The latest findings come as the European Hematology Association holds its annual meeting in Frankfurt.
The companies have been providing regular updates on patients treated with exa-cel in clinical trials, and the latest results show that many patients have lived for over a year without pain crises in the case of sickle cell and blood transfusions in the case of beta thalassemia, a time frame that helps validate the benefits of the treatment.
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