Genetix Biotherapeutics Inc.

Company Overview

Bluebird Bio Inc. has rebranded as Genetix Biotherapeutics after its recent acquisition by investment firms. The Carlyle Group and SK Capital Partners develop products in disease areas, including cerebral adrenoleukodystrophy, β-thalassemia, sickle cell disease, and multiple myeloma, utilizing cell and gene therapy technologies such as lentiviral gene addition and (megaTAL-enabled) gene editing. In oncology, the company focuses on developing novel T-cell-based immunotherapies, including CAR-T and T-cell receptor (TCR) therapies. It has a single operating segment that focuses on the research, development, and commercialization of gene therapies for severe genetic diseases. Zynteglo and SKYSONA are its flagship gene therapy products. Zynteglo is commercially available to patients in the U.S. through a network of 12 QTCs (defined as having a signed services agreement). Skysona is currently available through a network of three activated QTCs. Cell collection has been completed for two commercial SKYSONA patients as of March 2023. On March 16, 2023, the first commercial Skysona infusion was completed.

Company's Business Segments

• Therapies : Cerebral Adrenoleukodystrophy (CALD), Beta-thalassemia, Sickle Cell Disease