CHMP expresses support for bluebird bio's application for listing of Genenology Zynteglo

recently, bluebird bio announced that the EuropeanMedicines(http://Authority (EMA) has expressed its support for its application for approval (MAA) for gene therapy Zynteglo (formerly Known lentiGlobin)They recommended approval of this gene therapy for the treatment of patients over the age of 12 with blood transfusion-dependent beta-thalassemia (TDT) in non-beta 0/beta-0 genotypesTo survive, Patients with TDT require frequent blood transfusions throughout life and iron chelation therapyPatients with TDT are at risk of complications such as multiple organ failure due to iron overdoseAt present, the cure for TDT is an allogeneic hematopoietic stem cell transplant (HSCT), but HSCT is difficult to find the right HLA type, and there is a risk of complications including death due to treatment, transplant failure, transplant resistance to host disease and opportunistic infections, especially HSCT provided by non-direct blood relativesZyntegloZynteglo is a potential one-off cure gene therapyIt is through the genetic engineering of slow-virus-mediated genetic engineering of hematopoietic stem cells collected from the patient itself, the introduction of multiple copies of genetically modified genes that express normal beta-gloper protein, and then the modified cells are returned to the patient, thus achieving the potential cure of TDTThe FDA(http://awarded Zynteglo breakthrough therapy and fast-track eligibility for the treatment of TDTIn addition, Zynteglo has obtained THE EMA's PRIME qualificationThis CHMP support is based on the completed 1/2 Northstar (HGB-204)Test (http:// , 1/2 HGB-205 trial, the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) trials, and data obtained from the long-term follow-up test LTF-303 results show that as of September 2018, 80% of the 1/2 Northstar test subjects of the non-beta 0/beta 0 genotype achieved blood transfusion dependence: no blood transfusion stodport within 12 months, and a median of 38 months without blood transfusion Their total hemoglobin levels also reached more than 9 g/dL After 6 months, the average total hemoglobin level sitorized level situ in the Phase 3 Northstar-2 and Northstar-3 trials was 11.9 g/dL