CRISPR Gene Editing Therapy Human Clinical Trials Release Positive Data

Both beta thalassemia and sickle cell anemia are blood disorders caused by mutations in genes that encode hemoglobinSevere patients often need to be treated with regular red blood cell input, which not only inconveniences patients, but also long-term red blood cell input can cause side effects such as iron overloadCRISPRRS gene therapy CTX001 is an innovative gene-edited self-made hematopoietic stem cellCTX001 therapy uses CRISPR gene editing to increase the ability of hematopoietic stem cells obtained from the patient to express fetal hemoglobin (HbF), and then inject modified hematopoietic stem cells back into the patientHbF can compensate for functional defects in adult hemoglobin in patients, thereby relieving symptomsCTX001 improves the expression of fetal hemoglobin through gene editing (Photo: Source: 2) the interim results publishedinclude one TDT patient and one patient with severe SCDTDT patients needed an average of 16.5 blood transfusions per year before joining the trial9 months after receiving the CTX001 infusion, the patient no longer relied on blood transfusion, with a total hemoglobin level of 11.9 g/dL, of which the fetal hemoglobin level was 10.1 g/dLAnd 99.8% of red blood cells in the blood circulation express fetal hemoglobinthe performance of the first TDT patient after treatment with CTX001 (Photo: Supplied source, 2)group of SCD patients experienced an average of 7 vascular proctored visions (VOCs) per year prior to joining clinical trialsFor 4 months after receiving CTX001 infusion, the patient did not experience VOC again, with a total hemoglobin level of 11.3 g/dL, of which 46.6% was fetal hemoglobinMoreover, 94.7% of red blood cells in the blood circulation express fetal hemoglobin's performance after receiving CTX001 (Photo: Resources 2) Dr Samarth Kulkarni, CEO of CRISPR Therapeutics, said: "We are very encouraged by these preliminary dataThese data demonstrate the potential of our therapies to deliver meaningful benefits to patients after a one-time intervention We will continue to recruit patients to participate in research to accelerate the development of CRISPR/Cas9 therapy as a new type of conversion drug to treat serious diseases that are currently incurable References: crispR Therapeutics and VertEx Positive Safety and Efficacy Data From First First Patients Treated With With Crispal CRISPR/Cas9 Gene-Editing-Therapy CTX001® for HeSiproblobinopathies Retrieved 2019-11-19, from https://investors.vrtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-positive-safety-and CTX001 Clinical Data Update Retrieved November 19, 2019, from https://crisprtx.gcs-web.com/static-files/f1e96190-16d1-447c-a4f6-87cc28cc978f