Express One-time Gene Therapy Gets FDA Priority Review Qualification, Treats β-thalassemia

▎WuXi AppTec content team editor November 22, 2021, bluebird bio announced that the US FDA has granted priority review qualification for the biologics license application (BLA) for gene therapy betibeglogene autotemcel (beti-cel), which requires acceptance for treatment Beta thalassemia patients with routine red blood cell transfusion
.

The press release states that if approved, this will be the first in vitro gene therapy for hematopoietic stem cells approved by the FDA
.

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
.

In order to survive, the patient has to undergo blood transfusion for life
.

Although blood transfusions can temporarily alleviate the symptoms associated with severe anemia, including fatigue, weakness and shortness of breath, they cannot fundamentally treat the disease and may cause serious complications from iron overload and multiple organ damage
.

Chelation therapy for relieving iron overload is of limited effect, and there are compliance problems caused by drug tolerance
.

Beti-cel is a potential one-time treatment gene therapy
.

This gene therapy isolates hematopoietic stem cells from patients and uses viral vectors to introduce modified beta globulin genes that can perform normal functions
.

Subsequently, these patients will first receive chemotherapy to "cleanse" the bone marrow, and then introduce these gene-edited hematopoietic stem cells
.

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
.

Under ideal circumstances, patients no longer even need blood transfusion therapy
.

This therapy has previously obtained orphan drug designation and breakthrough therapy designation granted by the FDA, and has been approved by the European Union, under the trade name Zynteglo
.

Image source: The submission of this BLA of 123RF is based on the results of multiple clinical trials.
As of March 9, 2021, the trial results included 63 patients, of which two patients were followed up for 7 years and 8 patients were followed up The duration was at least 6 years, and 19 patients were followed up for more than 5 years
.

The latest data from the trial will be announced at the 63rd Annual Meeting of the American Society of Hematology (ASH) to be held from December 11 to 14, 2021
.

"For a long time, beta-thalassemia patients who rely on regular blood transfusions have had to endure a huge burden related to the disease
.

The unique role of beti-cel is to help patients produce normal or close to normal levels of adult hemoglobin, which is expected to eliminate their long-term blood transfusion and chelation.
require combined therapy
.

these treatment options are currently only temporarily alleviate symptoms of anemia, and may lead to serious health risks and reduced quality of life
.

"Ms.
Anne-Virginie Eggimann bluebird bio's chief regulatory officer, said," we look forward to working closely with the FDA , To provide this innovative therapy for patients in need
.

"References: [1] bluebird bio Announces FDA Priority Review of Biologics License Application for beti-cel Gene Therapy for Patients with β-thalassemia Who Require Regular Red Blood Cell Transfusions.
Retrieved November 22, 2021, from https://www.
businesswire.
com/news/home/20211122005845/en Disclaimer: WuXi AppTec's content team focuses on introducing global biomedical health research progress
.

This article is for information exchange purposes only.
The views in the article do not represent WuXi AppTec's position, nor do they represent WuXi AppTec's position Kant supports or opposes the views
in the
article .
This article is not a treatment plan recommendation
.

If you need treatment plan guidance, please go to a regular hospital
.