> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# bluebird bio BLA Accepted for Gene Therapy
- URL: https://www.fdaweb.com/bluebird-bio-bla-accepted-for-gene-therapy/
- Published: 2021-11-22T12:00:00.000Z
- Updated: 2026-09-14T17:23:09.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5150624

FDA has accepted for priority review a bluebird bio BLA for betibeglogene autotemcel (beti-cel), a gene therapy for adult, adolescent and pediatric patients with β-thalassemia across all genotypes who require regular red blood cell (RBC) transfusions. If approved, beti-cel would be a one-time treatment that addresses the underlying genetic cause of the disease, offering an alternative to regular RBC transfusions and iron chelation therapy. The agency has set a user fee review action target date of 5/20/2022.

The company says its therapy adds functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells to correct the deficiency, according to the company. In Phase 3 studies, 89% of evaluable patients across all ages and genotypes achieved transfusion independence, which is defined as no longer needing RBC transfusions for at least 12 months. “Adverse reactions considered related to beti-cel were uncommon and consisted primarily of non-serious infusion-related reactions that occurred on the day of infusion (e.g. abdominal pain, hot flush, dyspnea, tachycardia and non-cardiac chest pain) and cytopenias (e.g. thrombocytopenia, leukopenia and neutropenia),” bluebird says.