Bluebird's Zynteglo Gene Therapy Approved By Food and Drug Administration

By Kathryn Hardison

The U.S. Food and Drug Administration approved bluebird bio Inc.'s cell-based gene therapy on Wednesday 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 Zynteglo is the first cell-based gene therapy for the blood disorder.

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 kathryn.hardison@wsj.com

(END) Dow Jones Newswires

August 17, 2022 14:15 ET (18:15 GMT)

bluebird bio (NASDAQ:BLUE)
Historical Stock Chart
From Mar 2024 to Apr 2024