Prognosis
Crispr Surges as Gene Editing Shows Promise in Blood Disease
- Vertex and Crispr Therapeutics’ treatment used in two patients
- CTX001 halted complications from beta thalassemia, sickle cell
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A first-in-human study using the gene-editing tool Crispr has a company named after the technology and its partner, Vertex Pharmaceuticals Inc., claiming an early victory in treating two inherited blood disorders.
Crispr Therapeutics AG and Vertex said a pair of patients -- one suffering from beta thalassemia and the other with severe sickle cell disease -- saw benefits from one-time treatment with CTX001. While they’ve been followed for just nine months and four months, respectively, their painful flare-ups and once-routine need for blood transfusions stopped after getting the therapy. Both developed severe complications including infections that were deemed unrelated to the gene-editing treatment.