Gene therapy! Bluebird creature Lenti Globin has entered an official review in the European Union β for thalassemia

Bluebird Bio, a US gene therapy company, recently announced that the European Medicines Agency (EMA) has accepted applications for market approval (MAA) for gene therapy LentiGlobin to treat transfusion-dependent type β thrediotic anemia (TDT) and non-beta 0/beta genotype TDT adolescents and adult patients. The drug is a potential one-time gene therapy that can address the underlying genetic causes of TDT and is expected to revolution the clinical treatment of TDT. Previously, the EMA had granted LentiGlobin accelerated assessment eligibility, which would be shortened from seven months (210 days) to five months (150 days).
lentiGlobin is a gene therapy for thalassemia that implants genes that express normal hemoglobin β aki in vitro into hematopoietic stem cells taken from the patient and then returned to the patient through a lenvirus vector. On the EU side, the EMA has granted LentiGlobin the qualification for the treatment of TDT orphan drugs, fast track qualification, and priority drug qualification (PRIME). In the United States, the FDA has granted LentiGlobin the orphan drug and breakthrough drug qualification for TDT.
LentiGlobin MAA's submission is based on data from the completed Phase I/II Study Northstar (HGB-204), the ongoing Phase I/II HGB-205 study, and the Phase III Northstar-2 (HGB-207) study and the long-term follow-up study LTF-303. In April, data from The Northstar study and the HGB-205 study, published in the New England Journal of Medicine (NEJM), showed that most patients did not need blood transfusions for two years or more after receiving LentiGlobin infusions and showed normal hemoglobin levels.
David Davidson, chief medical officer of Bluebird Biology, said patients with blood transfusion dependence β thalt-thalassemia need frequent blood transfusions to save lives, but can also lead to complications, including organ failure due to iron overload. The acceptance of LentiGlobin MAA by the EMA is an important milestone that will move us towards providing patients with the goal of addressing the underlying causes of TDT. (Bio Valley)