FDA scuttles bluebird's accelerated plans for sickle cell gene therapy, as higher CMC bar forces 1-year delay
In yet another smudge on bluebird bio’s track record for execution, the biotech is pushing back the filing for its gene therapy in sickle cell disease by a year due to a new data requirement from the FDA.
With the setback, bluebird joins a league of gene therapy developers — from BioMarin to Voyager — that have been hampered by CMC issues. While somewhat unexpected, the regulators’ request highlights their stringency around manufacturing and quality control.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.