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CRISPR/Cas9 gene therapy CTX001 from Vertex Pharmaceuticals and CRISPR Therapeutics has been awarded the European Medicines Agency's Priority Drug (PRIME) title for the treatment of severe sickle cell disease (SCD).
Vascular obstruction and hemolysis are clinical characteristics of sickle cell disease, which can lead to recurrent painful episodes (formerly known as sickle cell distress), as well as a variety of serious organ system complications that can lead to lifelong disability and/or premature death.
based on advances in clinical trials, CTX001 has been awarded the U.S. Food and Drug Administration's (FDA) Advanced Therapy for Regenerative Medicine (RMAT), Fast Track and Orphan Drugs.
Vertex and CRISPR jointly developed the CTX001 under a commercial agreement.
prime title will speed up the development plan and evaluation of the CTX001.
CRISPR / Cas9 gene therapy CTX001 works by collecting the patient's own hematopoietic stem cells and ancestral cells from the blood, editing them using CRISPR/Cas9 techniques, and then sending the edited cells back to the patient as part of a stem cell transplant.
CRISPR Therapeutics has established strategic partnerships with many other leading pharmaceutical companies, including Bayer and ViaCyte.
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