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Vertex, Crispr Unveil More Encouraging Data From Sickle Cell, Thalassemia Gene Therapy Trials

  • Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) and Crispr Therapeutics AG (NASDAQ: CRSPhave announced new data from Phase 1/2 clinical trials evaluating CTX001 in beta-thalassemia and sickle cell disease.
  • Presenting data from two trials at the European Hematology Association annual meeting, the companies announced that three-month follow-up data of 22 patients with genetic blood disorders indicated a “consistent and sustained” response to the experimental drug CTX001.
  • All 15 patients with transfusion-dependent beta-thalassemia did not need further blood transfusions, and all seven with severe sickle cell disease were pain-free.
  • One thalassemia patient experienced a cerebellar hemorrhage less than three months after being treated.
  • The serious side effect was related to the busulfan conditioning that gene therapy patients undergo before receiving treatment.
  • Today, VRTX announced to discontinue VX-864 in alpha-1 antitrypsin deficiency as the treatment was unlikely to provide any clinical benefit.
  • Price Action: CRSP shares are up 2.43% at $126.71, while VRTX shares are down 8.62% at $198.08 during the market session on the last check Friday.

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