CRISPR gene editing therapy human clinical trials release positive data

CRISPR therapeutics and vertex pharmaceuticals announced yesterday that CRISPR / cas9 gene editing therapy ctx001 has yielded positive interim data in ongoing phase 1 / 2 clinical trials One patient with transfusion dependent β - thalassemia (TDT) and one patient with severe sickle cell anaemia (SCD) received ctx001, and both achieved the effect of stopping transfusion dependence This is the first human clinical trial to evaluate CRISPR gene editing therapy in the United States β - thalassemia and sickle cell anemia are blood diseases caused by mutations in the gene encoding hemoglobin Serious patients usually need to receive red blood cell input frequently for treatment, which not only brings inconvenience to patients, but also brings side effects such as iron overload CRISPR therapeutics gene therapy ctx001 is an innovative therapy for gene editing of autologous hematopoietic stem cells Ctx001 therapy uses CRISPR gene editing technology to increase the ability of hematopoietic stem cells obtained from patients to express fetal hemoglobin (HBF), and then inject the modified hematopoietic stem cells back into patients HbF can make up for the functional defect of adult hemoglobin, so as to alleviate the symptoms of patients ▲ ctx001 improves fetal hemoglobin expression through gene editing (picture source: reference [2]) Published interim results included a TDT patient and a severe SCD patient Patients with TDT need to receive an average of 16.5 blood transfusions per year before entering the trial Nine months after ctx001 infusion, the patient was no longer dependent on blood transfusion The total hemoglobin level was 11.9 g / dl, of which fetal hemoglobin level was 10.1 g / dl In addition, 99.8% of red blood cells in blood circulation express fetal hemoglobin ▲ the first TDT patient's performance after ctx001 treatment (picture source: reference [2]) Patients with SCD experienced an average of 7 vascular occlusive crises (VOC) per year before entering the clinical trial Within 4 months after ctx001 infusion, the patient did not experience VOC again, and the total hemoglobin level was 11.3 g / dl, 46.6% of which was fetal hemoglobin In addition, 94.7% of the red blood cells in the blood circulation expressed fetal hemoglobin ▲ the first SCD patient's performance after ctx001 treatment (picture source: reference [2]) "We are very encouraged by these preliminary data," said Dr samarth Kulkarni, chief executive of CRISPR therapeutics These data demonstrate the potential of our therapies to deliver meaningful benefits to patients after a single intervention We will continue to recruit patients to participate in research and accelerate the development of CRISPR / cas9 therapy as a new type of transforming drug to treat serious diseases that cannot be cured at present " reference material: [1] CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data From First Two Patients Treated With Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001® for Severe Hemoglobinopathies Retrieved 2019-11-19, from https://investors.vrtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-positive-safety-and [2] CTX001 Clinical Data Update Retrieved November 19, 2019, from https://crisprtx.gcs-web.com/static-files/f1e96190-16d1-447c-a4f6-87cc28cc978f A kind of