to give patients and their
families more bluebird days.
OUR ONE-TIME GENE THERAPY FOR SICKLE CELL DISEASE IS NOW FDA-APPROVED
Learn more about this treatment for sickle cell disease.
Setting the standard for gene therapy
For more than a decade, we’ve forged new paths for gene therapies and are improving and advancing at every step. We have the largest and deepest ex-vivo gene therapy data set in the world—driving the field forward, but we aren’t doing this alone. We learn from the realities of others. We engage in conversations that shape our actions and intentions. And we make sure to be good humans who are all-in every day.
Gene Addition Therapy: We’re building platforms with broad therapeutic potential because incremental improvements won’t do
Delivering more bluebird days for patients and their families requires a different approach to treating disease. In a rapidly advancing field, we’ve been leading the way for a decade. At bluebird, our proprietary lentiviral vector gene therapies are one-time treatments that are designed to deliver a functional copy of a gene to a patient’s own cells.
References:
Goswami R, Subramanian G, Silayeva L, et al. Gene therapy leaves a vicious cycle. Front Oncol. 2019;9:297.
Negre O, Eggimann A-V, Beuzard Y, et al. Gene therapy of the β-hemoglobinopathies by lentiviral transfer of the βA(T87Q)-globin gene. Hum Gene Ther. 2016;27:148-165.
National Institutes of Health. Genetics Home Reference. Help me understand genetics. Available at: https://medlineplus.gov/download/genetics/understanding/primer.pdf. Accessed April 2022.
Goswami R, Subramanian G, Silayeva L, et al. Gene therapy leaves a vicious cycle. Front Oncol. 2019;9:297.
Negre O, Eggimann A-V, Beuzard Y, et al. Gene therapy of the β-hemoglobinopathies by lentiviral transfer of the βA(T87Q)-globin gene. Hum Gene Ther. 2016;27:148-165.
National Institutes of Health. Genetics Home Reference. Help me understand genetics. Available at: https://medlineplus.gov/download/genetics/understanding/primer.pdf. Accessed April 2022.