Express one-time gene therapy takes an important step to treat beta thalassemia

▎The content team editor of WuXi AppTec 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
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It is used to treat beta thalassemia patients who need to receive routine red blood cell transfusion
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Previously, this gene therapy was granted a breakthrough therapy designation by the FDA
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The press release states that if approved, this will be the first in vitro gene therapy for hematopoietic stem cells approved by the FDA
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Transfusion-dependent beta thalassemia is a serious genetic disease
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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
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In order to survive, the patient has to undergo blood transfusion for life
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However, blood transfusion can only treat the symptoms, not the root cause, and due to the iron overload caused by blood transfusion, these patients are prone to multiple organ damage
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Therefore, these unfortunate patients urgently need safe and effective innovative therapies to alleviate their conditions
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Beti-cel brought by bluebird is such a new type of treatment
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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
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Subsequently, these patients will first receive chemotherapy to "cleanse" the bone marrow, and then introduce these gene-edited hematopoietic stem cells
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It is envisaged that these hematopoietic stem cells will continuously form hemoglobin variants with normal functions in the patient's body, greatly reducing the patient's need for blood transfusion
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Under ideal circumstances, patients no longer even need blood transfusion therapy
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Image source: The submission of the 123RF BLA is based on the results of multiple clinical trials.
As of March 9, 2021, the trial results included 63 patients, of which two patients have been followed up for 7 years and 8 patients have been followed up The duration was at least 6 years, and 19 patients were followed up for more than 5 years
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This therapy has been approved by the European Union under the trade name Zynteglo
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"This application submission brings us one step closer to our goal of providing transformative gene therapy for patients with beta thalassemia
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" said Andrew Obenshain, president of bluebird bio's serious genetic diseases.
"We look forward to working with the FDA to realize this one-time gene.
The potential of therapy
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"Reference: [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.
Retrieved September 21, 2021, from https : // Disclaimer: WuXi content teams to focus progress of biomedical research on global health introduction
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This article is only for information purposes, the text view does not represent the position of WuXi PharmaTech, It does not mean that WuXi AppTec supports or opposes the opinions
in the
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