Bluebird's Zynteglo Gene Therapy Approved by FDA -- Update
August 17 2022 - 2:43PM
Dow Jones News
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 kathryn.hardison@wsj.com
(END) Dow Jones Newswires
August 17, 2022 14:28 ET (18:28 GMT)
Copyright (c) 2022 Dow Jones & Company, Inc.
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