-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.
-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
-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-
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
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
– If granted Marketing Authorization, people ages 12 and older in Europe who have one F508del mutation and one minimal function mutation will for the first time be able to benefit from a medicine that treats the underlying cause of the disease – – People 12 years of age and older who have two
- After seven days of once-daily dosing with 200 mg of ALS-2200, genotype 1 patients with cirrhosis had a median 4.08 log 10 reduction in HCV RNA; among people with genotypes 3 or 4, there was a median 4.65 log 10 reduction in HCV RNA - - Data are consistent with previously reported ALS-2200
-Data provide proof-of-concept for use of VX-661 to further enhance CFTR function in people taking KALYDECO who have the F508del mutation and another mutation known to respond to KALYDECO alone- -Statistically significant improvements in lung function, as well as decreases in sweat chloride,
- For the first time, approximately 6,000 patients with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease– -12,000 people with one or two F508del mutations who are currently eligible for one of Vertex’s three other FDA -approved
