QuickView: Poised to advance gene therapy pipeline
bluebird is advancing a pipeline focused on gene therapy, a rapidly expanding area that has attracted significant industry interest. It aims to offer one-time, transformative therapies for rare diseases, which should support premium pricing, if commercialised. Its two clinical programmes, LentiGlobin and Lenti-D, have been granted orphan drug designation in the US and Europe. A CAR-T oncology collaboration with industry leader Celgene validates the technology’s promise.bluebird bio is a biotechnology company engaged in researching, developing, and commercializing potentially transformative gene therapies for severe genetic diseases and cancer. The company has built a product platform with a range of therapeutic potential in a variety of indications based on its lentiviral gene addition platform, gene editing and cancer immunotherapy capabilities. The company is developing LentiGlobin gene therapy for sickle cell disease; Lenti-D gene therapy for cerebral adrenoleukodystrophy; and in collaboration with Bristol-Myers Squibb, the company is developing bb2121 (idecabtagene vicleucel) and bb21217 product candidates as treatments for multiple myeloma.
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