CRISPR / Cas9 Therapy OTQ923 Treatment of Sickle Cell Disease: FDA Approves Clinical Trials
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Last Update: 2020-06-22
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Source: Internet
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Author: User
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Intellia Therapeutics announced Tuesday (March 31, 2020) that the FDA has approved the initiation of A Phase I/II clinical studies based on CRISPR/Cas9 cell therapy OTQ923, which is used to treat sickle cell disease (SCD)Andrew Schiermeier, Chief Operating Officer of Intellia, said: "This decision to move cell therapy into the clinic has the potential to significantly improve the lives of SCD patients." SCD is an inherited hemoglobin molecular disorder, when hemoglobin molecules are exposed to various environments, red blood cell hemoglobin polymerization, distortion and formation of sicklefetal hemoglobin (HbF) is a type of hemoglobin that is found mainly during the fetus, but has very little content in adulthood, however, a small amount of hbF remains in patients with sickle anemia and thalassemia, which has important benefits for alleviating clinical complications of anemiaAccording to Intellia, OTQ923 is based on CRISPR/Cas9 gene-edited hematopoietic stem cell (HSC) therapy, which highly expresses fetal hemoglobin (HbF) and sends gene-edited hematopoietic stem cells back into the patient, which is expected to reduce the risk of hemoglobin
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