Bluebird Bio Inc (NASDAQ: BLUE), a clinical-stage biotechnology company with a focus on developing and commercializing gene therapy products with a transformative potential for patients with genetic diseases and cancer. Blubird bio has become popular among investors and the scientific community after entail data from the HGB-205 study has been reported at the Annual Meeting of the American Society of Hematology (ASH) on December 8th 2014. The stock jumped more than 70% after the impressive data was revealed.
The bio-tech company has announced that interim data from the ongoing Phase 2/3 Study of the Efficacy and Safety of Ex Vivo Gene Therapy with Lentiviral Vector (Lenti-D) for the treatment of cerebral adrenoleukodystrophy (CALD) will be presented during the Clinical Trials plenary session on April 20, 2016 at the American Academy of Neurology (AAN) 2016 Annual Meeting.
The Starbeam Study is meant to explore Bluebird Bio’s Lenti-D gene therapy product candidate investigational gene therapy approach in boys up to 17 years of age who suffer from CALD.
The primary efficacy endpoint for the Starbeam Study is the proportion of subjects with no major functional disabilities at 24 months after treatment. There are six specific functional disabilities associated with the Neurological Function Score (NFS) that have a severe negative impact on patients’ lives: loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence and complete loss of voluntary movement.
Gene therapy is a revolutionary endeavor, and if successful may change the way we treat several severe diseases including Sickle Cell Anemia. Gene therapy works by genetically modifying a patient’s own cells, adding a functional copy of the gene of that was damaged. According to Bluebird Bio, “We have extensive expertise in viral vector design and manufacturing and transduction, which we have developed into a potent gene therapy platform with potentially broad applications in a wide variety of indications with significant medical needs.”
Bluebird Bio is in a good financial position. The company has $61 million in debt, and $518 million cash was indicated on the attest earnings report. The stock is currently trading about 75% below its 52 week high levels.