FDA lets bluebird resume trials for sickle cell gene therapy after cancer scare, but big questions linger over field
Bluebird bio announced that the FDA has lifted its holds on clinical trials for their gene therapies for sickle disease and beta-thalassemia, ending a 4-month saga that began after the biotech reported that two patients had come down with cancer and a cancer-like condition.
Regulators were concerned that the cases may have been triggered by the virus bluebird uses to deliver a healthy gene for hemoglobin into patients with rare blood disorders, as had happened in a 2004 gene therapy trial that relied on a similar virus. Bluebird largely allayed those concerns in March, when they released a genetic analysis showing that the virus didn’t interfere with the patient’s DNA in a way likely to trigger cancer, but experts say the cases have broadly raised concerns about the risks any of the more than half dozen of sickle disease gene therapies now in clinical development may pose.
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