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I have made a few bluebird houses and they had bluebirds occupy them, but that is not the reason this board is starting up. bluebird bio Inc. is a nascent biotech company that has the potential to be a disruptive force in this space. Here is the overview of BLUE from their website: Gene Therapy - True Personalized Medicine "Our objective is to develop and commercialize a next generation of products based on the transformative potential of gene therapy as a one-time treatment option for patients with severe genetic and orphan diseases. Central to this effort is a collective determination within our company to provide these patients with hope for a better life in the face of limited or no long-term safe and effective treatment options."    "Our approach represents a highly personalized, important new therapeutic modality for patients with severe genetic and orphan diseases with significant unmet medical need. We have clinical-stage programs in development for childhood cerebral adrenoleukodystrophy, beta-thalassemia and sickle cell disease, as well as a preclinical oncology program in the chimeric antigen receptor (CAR) T cells field under a collaboration with Celgene Corporation." "Our gene therapy process involves inserting genetic material into the patient's own cells ex vivo, then re-introducing the cells to the patient. For beta-thalassemia, sickle cell disease and adrenoleukodystrophy, we insert a functional copy of the malfunctioning gene into the patient's hematopoietic stem cells (HSCs), with the goal of genetically modifying a patient's own cells to fundamentally correct or address the genetic basis underlying a disease. In oncology, the gene therapy process targets a different cell type, called T cells. In this case, genetic sequences are inserted into a patient's own T cells and are intended to program the T cells to specifically recognize and attack cancer cells. In all cases, gene therapy has the potential to change the way patients are treated by correcting the underlying genetic defect that is the cause of their disease or by providing additional functionality that can eliminate the disease, rather than offering solutions that only address their symptoms. Accordingly, we believe gene therapy has the potential to provide transformative disease modifying effects with life-long clinical benefits based on a single therapeutic administration." BLUE had its IPO priced at $17/share, and was a darling on Wall Street as it opened on its IPO day, at $25.50 and closed that day at $26.91. The stock has recently dropped back to the IPO price area. investor.bluebirdbio.com investor.bluebirdbio.com I was drawn to BLUE based on its CAR T technology, developed by Carl June, MD, a specialist in T cell biology and lymphocyte activation at the Perelman School of Medicine at the University of Pennsylvania. This is an immunotherapy approach, and here is a lecture on engineering T cells to cure cancer. youtube.com Here is a presentation by the CEO Nick Leschly at TEDxBoston on gene therapy. youtube.com | ||||||||||||||
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