Post by buffettlike on Oct 11, 2018 11:50:09 GMT -5
CRISPR Therapeutics and Vertex Announce FDA Has Lifted the Clinical Hold on the Investigational New Drug Application for CTX001 for the Treatment of Sickle Cell Disease
ZUG, Switzerland and BOSTON and CAMBRIDGE, Mass., Oct. 10, 2018 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold and accepted the Investigational New Drug application (IND) for CTX001 for the treatment of sickle cell disease (SCD). CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies.
In addition to the acceptance of the IND announced today, CRISPR and Vertex previously announced that they had obtained approvals of Clinical Trial Applications for CTX001 in multiple countries outside the U.S. for both β-thalassemia and SCD. The companies remain on track to initiate a Phase 1/2 clinical study in SCD by the end of 2018 and are currently enrolling patients with transfusion dependent β-thalassemia in a Phase 1/2 trial in β-thalassemia in Europe.
ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-fda-has-lifted-clinical#