Release date: 2017-12-11
On December 7, CRISPR Therapeutics announced the submission of a CTA clinical trial application for CTX001. CTX001 is a CRISPR-based gene editing therapy developed by CRISPR Therapeutics and Vertex Pharmaceuticals for the treatment of severe beta thalassemia and sickle cell anemia. Once the CTA clinical trial application is approved, it will be the first commercial CRISPR gene editing therapy to enter the clinical trial phase.
CTX001 through the genetic modification and reinfusion of the patient's autologous hematopoietic stem cells, so that the patient's red blood cells high expression of fetal hemoglobin HbF, improve the patient's red blood cell oxygen supply, to achieve therapeutic purposes. HbF is a hemoglobin that can carry a lot of oxygen, usually expressed at birth, and replaced with adult hemoglobin in adulthood. If the patient has problems with hemoglobin in adults, serious diseases such as β-thalassemia and sickle cell anemia may occur. By high expression of HbF, it can replace adult hemoglobin and supply oxygen to the body.
CTX001 clinical trials in Europe are expected to begin in 2018 to assess the efficacy and safety of CTX001 in adult beta thalassemia patients. At the same time, CRISPR Therapeutics plans to apply for NDA new drug application to the US FDA in 2018, and use CTX001 for clinical trials of sickle cell anemia.
Source: Medical Maike
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