Ten patients treated with a CRISPR-based gene-editing therapy for the inherited blood disorders sickle cell disease and beta-thalassemia have shown a consistent and sustained response with manageable side effects, according to interim results from two clinical trials reported Saturday.
Vertex Pharmaceuticals and CRISPR Therapeutics are jointly developing the one-time gene-editing treatment called CTX001. After being given the therapy by infusion, all patients in both studies have been free from symptoms of the diseases and have not needed blood transfusions. Although follow-up is still relatively short, the encouraging results raise hope that one-time cures for both blood disorders might be possible.
CTX001 is the first and most advanced effort to use the Nobel Prize-winning CRISPR/Cas9 technology to provide functional cures for inherited diseases.
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