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Main focus: Gene therapies and gene editing for the treatment of genetic diseases
Company stage: Clinical
Diseases (gene editing): Haemophilia and other severe genetic diseases
Genome editing tool: megaTAL
Funding stage: Public
Location: Cambridge, MA, USA
Gene editing partnerships: Novo Nordisk
bluebird bio develops novel gene and cancer therapies. The company is developing its proprietary megaTAL gene-editing technology, although it still has to disclose its first pre-clinical candidate derived from this. The company has a collaboration with Novo Nordisk to jointly develop next generation in vivo genome-editing treatments for genetic diseases, including haemophilia.