bluebird bio
bluebird bio is dedicated to developing and delivering gene therapies for severe genetic diseases with limited or no treatment options. They focus on innovative gene therapy technologies, including their proprietary lentiviral vector platform, to provide transformative benefits and hope for patients and their families. With over a decade of expertise and more than 200 patients treated, bluebird bio aims to set the standard in gene therapy, addressing diseases at their root cause and improving lives.
Industries
Nr. of Employees
large (251-1000)
bluebird bio
Somerville, Massachusetts, United States, North America
Products
One-time ex vivo lentiviral gene therapy for sickle cell disease (FDA-approved Dec 8, 2023)
A one-time autologous hematopoietic stem cell gene addition therapy using a lentiviral vector, approved for treatment of sickle cell disease.
One-time ex vivo lentiviral gene therapy for transfusion-dependent beta-thalassemia (FDA-approved Aug 17, 2022)
An autologous hematopoietic stem cell gene addition therapy using a lentiviral vector, approved for transfusion-dependent beta-thalassemia.
One-time ex vivo lentiviral gene therapy for cerebral adrenoleukodystrophy (FDA-approved Sep 16, 2022)
An autologous hematopoietic stem cell gene addition therapy using a lentiviral vector, approved for cerebral adrenoleukodystrophy (CALD).
One-time ex vivo lentiviral gene therapy for sickle cell disease (FDA-approved Dec 8, 2023)
A one-time autologous hematopoietic stem cell gene addition therapy using a lentiviral vector, approved for treatment of sickle cell disease.
One-time ex vivo lentiviral gene therapy for transfusion-dependent beta-thalassemia (FDA-approved Aug 17, 2022)
An autologous hematopoietic stem cell gene addition therapy using a lentiviral vector, approved for transfusion-dependent beta-thalassemia.
One-time ex vivo lentiviral gene therapy for cerebral adrenoleukodystrophy (FDA-approved Sep 16, 2022)
An autologous hematopoietic stem cell gene addition therapy using a lentiviral vector, approved for cerebral adrenoleukodystrophy (CALD).
Services
Personalized patient support throughout treatment journey
Clinical navigation and individualized support services for patients undergoing gene therapy treatment.
Collaboration and licensing partnerships
Business development engagements to partner or license technologies for expanding gene therapy applications and platform capabilities.
Grants and sponsorships for patient and scientific programs
Provision of restricted grants and sponsorship funding to nonprofit advocacy organizations and scientific/educational programs aligned with company focus areas.
Access coordination via Qualified Treatment Center network
Coordination of patient access to approved therapies through a network of trained hospital centers and guidance on treatment pathways and clinical site contacts.
Clinical trial enrollment and investigator contact support
Support for patients and clinicians seeking participation in investigational studies, including contact pathways to inquire about trials.
Personalized patient support throughout treatment journey
Clinical navigation and individualized support services for patients undergoing gene therapy treatment.
Collaboration and licensing partnerships
Business development engagements to partner or license technologies for expanding gene therapy applications and platform capabilities.
Grants and sponsorships for patient and scientific programs
Provision of restricted grants and sponsorship funding to nonprofit advocacy organizations and scientific/educational programs aligned with company focus areas.
Access coordination via Qualified Treatment Center network
Coordination of patient access to approved therapies through a network of trained hospital centers and guidance on treatment pathways and clinical site contacts.
Clinical trial enrollment and investigator contact support
Support for patients and clinicians seeking participation in investigational studies, including contact pathways to inquire about trials.
Expertise Areas
- Ex vivo lentiviral gene therapy development
- Autologous hematopoietic stem cell gene therapy
- Clinical trial management (Phases I–IV) and long-term follow-up
- Regulatory submissions and post-marketing commitments
Key Technologies
- Lentiviral vector-based ex vivo gene addition
- Autologous hematopoietic stem cell modification
- Ex vivo cell processing workflows
- Long-term clinical outcome monitoring