Ahead of 2-day adcomm, FDA sounds positive on one bluebird gene therapy but questions remain for another
In what may be make-or-break meetings for bluebird bio, the FDA’s cell and gene therapy advisory committee will meet Thursday and Friday to discuss the company’s two potential new gene therapies, with the agency signaling a likely green light for one to treat a rare blood disorder and questions for the other.
The agency signaled in briefing documents released Tuesday that it’s ready to approve the company’s likely expensive beti-cel gene therapy to treat β-thalassemia for those who require regular blood transfusions.
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