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First CRISPR gene-editing filings to be accepted for review by FDA - FDA grants Priority Review for severe sickle cell disease (SCD) and Standard Review for transfusion-dependent beta thalassemia (TDT)
Both trials met the primary and key secondary endpoints at the pre-specified interim analysis - Data continue to demonstrate transformative and durable benefit - Safety profile consistent with busulfan
-Initiated clinical trials for next generation CAR T candidates, CTX112™ targeting CD19+ B-cell malignancies and CTX131™, targeting CD70+ solid tumors- -Enrollment... | May 8, 2023
-Regulatory submissions complete for exagamglogene autotemcel , formerly known as CTX001™, in the U.S. for transfusion-dependent beta thalassemia and severe sickle cell disease - -EU and U.K.... | May 8, 2023
A rare, life-threatening genetic disease - and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, cell and... | April 3, 2023