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, May 14, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that new data from two ongoing Phase 1/2 clinical trials of the CRISPR/Cas9 gene-editing therapy CTX001 in
BOSTON --(BUSINESS WIRE)--Jun. 3, 2020-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Diana McKenzie has been appointed to its board of directors as an independent director. Ms. McKenzie is a senior technology leader and innovator with deep experience across pharma,
- KALYDECO ® (ivacaftor) is the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual function mutation, in children as young as 6 months of age - LONDON --(BUSINESS WIRE)--Jun.
-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-
- Vertex Foundation Announces $1.5 Million Gift to Boston University’s New Center for Antiracist Research - -Biotechnology-focused curriculum established in partnership with Year Up- -Additional commitments made to STEAM education- BOSTON --(BUSINESS WIRE)--Jun.
– 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
-CF patients in England will be among the first in Europe to benefit from access to KAFTRIO ® , if the medicine is approved by the European Commission - LONDON --(BUSINESS WIRE)--Jun. 30, 2020-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it has expanded its
BOSTON --(BUSINESS WIRE)--Jul. 16, 2020-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its second-quarter 2020 financial results on Thursday, July 30, 2020 after the financial markets close. The company will host a conference call and webcast at 5:30 p.m. ET .
-Phase 3 study met primary endpoint and all secondary endpoints- -Study is a U.S. post-marketing commitment and will be submitted to FDA- -Data also will be submitted to the European Medicines Agency to support indication expansion of the EU label following triple combination approval- BOSTON
