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# bluebird bio BLA for Sickle Cell Gene Therapy
- URL: https://www.fdaweb.com/bluebird-bio-bla-for-sickle-cell-gene-therapy/
- Published: 2023-04-24T12:00:00.000Z
- Updated: 2026-09-14T18:27:11.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5154284

bluebird bio has submitted for priority review a BLA for lovotibeglogene autotemcel (lovo-cel), a gene therapy for patients with sickle cell disease ages 12 years and older who have a history of vaso-occlusive events. The submission is based on data from 36 patients in the HGB-206 Group C cohort with a median 32 months of follow-up and two patients in the HGB-210 study with 18 months of follow-up each, according to the company. It also includes safety data from 50 patients treated across the entire lovo-cel program, including six patients with six or more years of follow-up. Previously, the agency granted the products an orphan drug designation, fast track designation, regenerative medicine advanced therapy designation, and rare pediatric disease designation.

The gene therapy is a one-time treatment that is designed to add functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells, the company says. Once patients have the targeted gene, their red blood cells can produce anti-sickling hemoglobin that can reduce sickled red blood cells, hemolysis, and other complications, it says.