Vertex and CRISPR Therapeutics Announce Priority Medicines (PRIME) Designation Granted by the European Medicines Agency to CTX001™ for Transfusion-Dependent Beta Thalassemia
26 Apr 2021
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of transfusion-dependent beta thalassemia (TDT).
CRISPR
17 Nov 2020
Gene editing, a group of technologies that give scientists the ability to change an organism's DNA, is of vital importance in cell and gene therapy. CRISPR is a revolutionary gene editing technology which has become the hottest research tool in the field of biological sciences in just a few years since its introduction in 2012. However, people may still wonder if there are any other types of gene editing technologies.
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