bluebird bio
bluebird bio works on 3 rare diseases tracked on Trial Friend, including Cerebral Adrenoleukodystrophy, Sickle Cell Disease, Thalassemia.
bluebird bio is a gene therapy company with 3 FDA-approved products: Zynteglo (betibeglogene autotemcel) for transfusion-dependent beta thalassemia, Lyfgenia (lovotibeglogene autotemcel) for sickle cell disease, and Skysona (elivaldogene autotemcel) for cerebral adrenoleukodystrophy. The company was acquired by Carlyle Group and SK Capital Partners in a take-private deal completed June 2025 after facing commercial challenges with its gene therapy launches.
Focus areas at bluebird bio
As a rare disease specialist, bluebird bio has active clinical trial programs and drug development efforts across 3 rare diseases, including Cerebral Adrenoleukodystrophy, Sickle Cell Disease, Thalassemia. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by bluebird bio, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
bluebird bio is headquartered in Somerville, United States, founded in 2010, publicly traded under the ticker symbol BLUE, a subsidiary of Carlyle Group / SK Capital Partners (acquired June 2025). The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Active Clinical Trials
FDA-Approved Drugs
Rare Disease Focus Areas (3)
Diseases targeted by bluebird bio's clinical trial and drug development programs
Cerebral adrenoleukodystrophy is the most severe form of X-linked adrenoleukodystrophy, caused by mutations in the ABCD1 gene that impair breakdown of very-long-chain fatty acids. These accumulate in ...
Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion. Thi...
Thalassemia is an inherited blood disorder where reduced or absent production of one type of hemoglobin chain causes severe anemia, organ damage from iron overload, and bone abnormalities. Severe form...
Patient Resources
Organizations and resources related to bluebird bio's rare disease focus areas
Frequently Asked Questions About bluebird bio
Common questions about bluebird bio's rare disease programs, clinical trials, and treatments.