Benzinga | Sep 22, 2020 09:05AM EDT

CRISPR Therapeutics And Vertex Pharmaceuticals Announce Priority Medicines Designation Granted By European Medicines Agency To CTX001 For Treatment Of Sickle Cell Disease

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Sept. 22, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated(NASDAQ:VRTX) 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 severe sickle cell disease (SCD).



PRIME is a regulatory mechanism that provides early and proactive support to developers of promising medicines, to optimize development plans and speed up evaluations so these medicines can reach patients faster. The goal of PRIME is to help patients benefit as early as possible from innovative new therapies that have demonstrated the potential to significantly address an unmet medical need. PRIME designation was granted based on clinical data from CRISPR and Vertex's ongoing Phase 1/2 trial of CTX001 in patients with severe SCD.