ZUG, Switzerland and CAMBRIDGE and BOSTON, Mass. – April 16, 2019 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the
ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Jan. 04, 2019 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the
-Beta thalassemia: Two patients are transfusion independent at 5 and 15 months after CTX001 infusion; data demonstrate clinical proof-of-concept for CTX001 in transfusion-dependent beta thalassemia- -Sickle cell disease: Patient is free of vaso-occlusive crises at 9 months after CTX001 infusion-
-Two patients treated with CTX001 successfully engrafted and demonstrated an initial safety profile consistent with myeloablative busulfan conditioning and autologous hematopoietic stem cell transplant- -Beta thalassemia: Patient is transfusion independent with total hemoglobin level of 11.9 g/dL
-First patient infused with CTX001 in a Phase 1/2 clinical trial for patients with beta thalassemia- -First patient enrolled in a Phase 1/2 clinical trial of CTX001 for patients with sickle cell disease- ZUG, Switzerland and CAMBRIDGE and BOSTON, Mass., Feb.
CTX001 has received Orphan Drug Designation from the U.S. Food and Drug Administration for transfusion-dependent beta thalassemia and from the European Medicines Agency for sickle cell disease and transfusion-dependent beta thalassemia ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 11,
ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Sept. 22, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Inco r porated (Nasdaq: VRTX) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an
- Beta thalassemia: All seven patients were transfusion independent with 3 to 18 months of follow-up after CTX001 infusion - - Sickle cell disease: All three patients were free of vaso-occlusive crises with 3 to 15 months of follow-up after CTX001 infusion - - Nineteen patients have been dosed with
BOSTON and ZUG, Switzerland and CAMBRIDGE, Mass. , May 30, 2018 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Investigational New Drug
