FDA Lifts Hold on Bluebird Bio’s Gene Therapy Trials

The FDA has lifted clinical holds on studies evaluating Bluebird Bio’s LentiGlobin gene therapy for sickle cell disease (SCD) as well as trials of a treatment for a blood disorder that uses the same viral vector.

In February, the agency ordered the company to pause several studies evaluating LentiGlobin for SCD after a patient developed acute myeloid leukemia (AML) five years after being treated with the therapy. The company later concluded it was very unlikely the cancer was caused by the treatment, which apparently satisfied the FDA reviewers.

Bluebird Bio also halted two phase 3 studies of betibeglogene autotemcel (beti-cel) for patients with transfusion-dependent beta thalassemia, because the product is produced using the same lentiviral vector as LentiGlobin. Because the company resolved the agency’s concerns regarding LentGlobin, the beti-cel trials were also given a green light to restart.

The company will now resume the studies and seek regulatory submissions for SCD and beta-thalassemia, said Andrew Obenshain, Bluebird Bio’s president of severe genetic diseases.