bluebird bio Soars as FDA Adcom Recommends Second Gene Therapy

After two days of eagerly anticipated discussion, the U.S. Food and Drug Administration’s Cell, Tissue and Gene Therapies Advisory Committee voted on bluebird bio’s two lentiviral vector (LVV) gene therapies.

On bluebird’s betibeglogene autotemcel (beti-cel) for beta-thalassemia, the advisory committee was unanimous, voting 13 (yes) to 0 (no) on the question, “Do the benefits of beti-cel outweigh the risks for the treatment of subjects with transfusion-dependent beta-thalassemia?”