Rare disease drugmaker · part of Carlyle Group / SK Capital Partners (acquired June 2025) · based in Somerville, United States · founded 1992 · genetixbiotx.com
bluebird bio works on 3 rare diseases we track, including Cerebral Adrenoleukodystrophy, Sickle Cell Disease and 1 more. It has 6 active clinical trials, and 2 are looking for volunteers now. It also has 3 FDA-approved rare disease drugs.
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. Funds managed by Carlyle and SK Capital Partners took the company private on June 2, 2025, after commercial challenges with its gene therapy launches.
bluebird bio pipeline
Each row lists the diseases bluebird bio is studying at that stage, from the ones closest to approval down to the earliest work.
These are bluebird bio trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
2 trials looking for volunteers now
Looking for volunteersHasn't posted an update in over a yearNCT06224413
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major functional disability (MFD)-free survival over time in participants with more advanced early active CALD. All enrolled participants with CALD treated with eli-cel in the post-marketing setting will be followed in this study for 15 years. No investigational drug product will be administered in this study. This study will enroll 120 participants with CALD treated with eli-cel in the post-marketing setting. A subpopulation of 24 participants with more advanced early active CALD will be specifically enrolled as required by the US FDA as a condition of accelerated approval and will be considered as a separate cohort for effectiveness outcomes.
Looking for volunteersHasn't posted an update in over a yearNCT06271512
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
TestsNo Intervention
For Beta-Thalassemia
The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study ALD-104).
After completing a parent clinical study (approximately 2 years), eligible participants will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own disease. In gene therapy, we introduce new genetic material in order to fix or replace the patient's disease gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of graft versus host disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. To introduce new genetic material into the patient's own blood stem cells we use a modified version of a virus (called a 'vector') that efficiently inserts the "correcting" genetic material into the cells. The vector is a specialized biological medicine that has been formulated for use in human beings.
Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. The investigators have discovered a gene that is very important in controlling the amount of HbF. Decreasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, specifically the amount in red blood cells where sickle hemoglobin causes damage to the cell, and therefore potentially cure or significantly improve the condition. The gene we are targeting for change in this study that controls the level of fetal hemoglobin is called BCL11A.
In summary, the advantages of a gene therapy approach include: 1) it can be used even if the patient does not have a matched donor available; 2) it may allow a reduction in the amount of chemotherapy required to prepare the patient for the transplant; and 3) it will avoid certain strong medicines often required to prevent and treat GVHD and rejection. Our lab studies with normal mice, mice that have a form of SCD, and with cells from the bone marrow of SCD patients who have donated bone marrow for research purposes show this approach is very effective in reducing the amount of sickle hemoglobin in red cells. Our pilot trial testing this approach in 10 patients with SCD has shown that the treatment has not caused any unexpected safety problems, and that it increases HbF within the red blood cells. Our goal is to continue to test whether this approach is safe, and whether using gene therapy to change the expression of BCL11A will lead to decreased episodes of vaso-occlusive crisis pain in people with SCD.
Underway, not taking new volunteersHasn't posted an update in over a yearNCT02633943
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
TestsSafety and efficacy assessments
For Transfusion-dependent Beta-Thalassemia
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent clinical studies. After completing the parent clinical studies (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.
This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD).
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.
How common: 1 in 20,000 males worldwide; cerebral form develops in approximately 35-40% of boys with X-ALD
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.
How common: About 100,000 Americans with SCD; about 1 in 365 Black or African American births and 1 in 16,300 Hispanic American births
Thalassemia is an inherited blood disorder where reduced or absent production of one type of hemoglobin chain causes anemia, from mild or none in carriers to severe in the major forms, where it can...
How common: About 1 in 100,000 people have symptoms of beta thalassemia, according to NORD, most often people with Mediterranean, Middle Eastern, African or Asian roots
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. bluebird bio's own site is genetixbiotx.com, and our help paying for treatment page lists drugmaker programs and charity funds.
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. Funds managed by Carlyle and SK Capital Partners took the company private on June 2, 2025, after commercial challenges with its gene therapy launches. On September 18, 2025 it renamed itself Genetix Biotherapeutics, close to its 1992 founding name, Genetix Pharmaceuticals.
bluebird bio has active programs across 3 rare diseases: Cerebral Adrenoleukodystrophy, Sickle Cell Disease, Thalassemia. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Yes. bluebird bio is a rare disease specialist headquartered in Somerville, United States, founded in 1992. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Cerebral Adrenoleukodystrophy, Sickle Cell Disease, Thalassemia. It operates as part of Carlyle Group / SK Capital Partners (acquired June 2025).
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by bluebird bio, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
bluebird bio has 3 FDA-approved drugs with a rare disease use that we track, as of November 2026.
Lyfgenia: Genetix CARES (formerly my bluebird support), 1-833-888-6378. Offers travel help and insurance help. Checked September 24, 2026.
Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.
Eligibility is decided by each program, so call the number listed before a prescription is filled.
bluebird bio was founded in 1992 and is headquartered in Somerville, United States. The company has been active for 34 years. It is a subsidiary of Carlyle Group / SK Capital Partners (acquired June 2025). As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.