US clinical-stage biotech firm bluebird bio (Nasdaq: BLUE) is advancing a pipeline focused on gene therapy, a rapidly expanding area that has attracted significant industry interest, note analysts at Edison Equity Research in a note on the company.
bluebird aims to offer one-time, transformative therapies for rare diseases, which should support premium pricing, if commercialized. Its two clinical programs, LentiGlobin and Lenti-D, have been granted orphan drug designation in the USA and Europe. A CAR-T oncology collaboration with industry leader Celgene validates the technology’s promise.
Promising early clinical data for orphan programs
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