By Kathryn Hardison


The U.S. Food and Drug Administration approved bluebird bio Inc.'s cell-based gene therapy called Zynteglo.

The therapy is designed for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusion. The FDA said Wednesday that Zynteglo is the first cell-based gene therapy for the blood disorder.

"We are ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care," Chief Executive Andrew Obenshain said.

Mr. Obenshain said the approval follows more than a decade of research and clinical development.

Zynteglo is a one-time gene therapy product administered as a single dose. The agency said that each dose of Zynteglo is a customized treatment created using the patient's own cells that are genetically modified to produce functional beta-globin.

Zynteglo's safety and effectiveness were established in two multicenter clinical studies that included both adult and pediatric patients. The most common reactions associated with Zynteglo included reduced platelet and other blood cell levels, as well as symptoms like vomiting, alopecia, cough, headache, rash and more.


Write to Kathryn Hardison at


(END) Dow Jones Newswires

August 17, 2022 14:28 ET (18:28 GMT)

Copyright (c) 2022 Dow Jones & Company, Inc.
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