IPO bluebird bio, Inc. (BLUE) is a clinical-stage biotechnology company focused on treating severe genetic and orphan diseases using gene therapy. Many diseases have a genetic aspect whereby a mutated gene linked to a disease is passed down from generation to generation. Gene therapy has the potential to correct the underlying genetic defect that is the cause of the disease, rather than offering solutions that only address the symptoms. Bluebird and their scientific collaborators have generated what they believe is human proof-of-concept data for their gene therapy platform in two underserved diseases, each of which has been granted orphan drug status by U.S. and European regulatory authorities.
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