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Today, bluebird bio announced that it has completed the submission of a rolling biologics license application (BLA) for gene therapy betibeglogene autotemcel (beti-cel) to the US FDA
.
It is used to treat beta thalassemia patients who need to receive routine red blood cell transfusion
Transfusion-dependent beta thalassemia is a serious genetic disease
.
Due to mutations in the gene encoding β-globin, the level of hemoglobin in the patient's body will significantly decrease or even be missing
Beti-cel brought by bluebird is such a new type of treatment
.
Specifically, this gene therapy isolates hematopoietic stem cells from patients and uses viral vectors to introduce modified beta globulin genes that can perform normal functions
The submission of this BLA is based on the results of multiple clinical trials.
As of March 9, 2021, the trial results include 63 patients, of which two patients have been followed up for up to 7 years and 8 patients have been followed up for at least 6 years , 19 patients were followed up for more than 5 years
.
This therapy has been approved by the European Union under the trade name Zynteglo
Reference materials:
[1] bluebird bio Submits Biologics License Application (BLA) to FDA for betibeglogene autotemcel (beti-cel) Gene Therapy for Patients With β-thalassemia Who Require Regular Red Blood Cell Transfusions.
(The original text has been deleted)
