Bluebird bio, specialists in developing gene therapies for genetic diseases and immunotherapies for cancer, have announced that they will present data from their ongoing Phase 2/3 Starbeam study at the American Academy of Neurology (AAN) 2016 Annual Meeting.
The gene therapy product candidate has been developed with a lentiviral (Lenti-D) vector which will function as a potential treatment for cerebral adrenoleukodystrophy (CALD), a neurodegenerative disease affecting young males that can be fatal if untreated. The only current therapy that has proven affective are allogeneic hematopoietic stem cell (HSC) transplants but is not without risks. These involve graft rejection and transplant mortality. The product candidate is made up of the patient’s own HSCs which should alleviate the issues associated with current treatment methods.
The Starbeam Study is designed to assess the safety and efficacy of the gene therapy in young males, up to the age of 17, diagnosed with CALD. The onset of CALD is brought on by the absence of a crucial protein required to break down very long chain fatty acids, which builds up in the CNS and results in the neurodegeneration associated with the disease. The therapy involves the transplantation of a patient’s own modified stem cells carrying a functional copy of the gene encoding the protein that breaks down the fatty acids. The primary efficacy endpoint of this study will be the proportion of patients that have no major functional disabilities 24 months after infusion.
Source: bluebird bio to Present Clinical Data on Lenti-D in CALD in Plenary Session at AAN 2016 Annual Meeting; Press Release