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One-time gene therapy receives FDA priority review qualification for the treatment of beta thalassemia

 Last Update: 2021-12-01
 Source: Internet
 Author: User

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clone gene

mri therapy

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On November 22, 2021, bluebird bio announced that the U.

Transfusion-dependent beta thalassemia is a serious genetic disease

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

The submission of this BLA is based on the results of multiple clinical trials.

Reference materials:

[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.

(The original text has been deleted)

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