Medical Professionals

our clinical trials

We are leading the gene therapy revolution. Our integrated product platforms encompass gene therapy, cancer immunotherapy and genome editing – providing us with the potential to treat, and hopefully cure, a broad range of serious diseases.

We designed the eligibility criteria for each of our current clinical trials to evaluate investigational drug products in a specific population of patients with cerebral adrenoleukodystrophy and adults and adolescents with transfusion-dependent β-thalassemia (also known as β-thalassemia major) and severe sickle cell disease (SCD). This is so we can gather information on the safety and effectiveness of the respective investigational drug products. Until there are adequate data demonstrating safety and efficacy, access to the investigational gene therapy is limited to the clinical trials (ALD-102, HGB-204, HGB-205, HGB-206, HGB-207 and CRB-401) for patients meeting eligibility criteria!

For more information about bluebird bio-sponsored clinical trials, please contact us at

Learn more about our ongoing clinical trials: