Seattle Children’s, Biotech Collaborate on Gene Editing

Mountain bluebird (Elaine Wilson, Wikimedia Commons)

11 September 2015. The biotechnology company bluebird bio and Seattle Children’s Research Institute are partnering on gene editing technologies to treat inherited pediatric diseases. Their agreement calls for bluebird bio — the name is spelled in all lower-case characters — based in Cambridge, Massachusetts and Seattle — to provide $3 million for research on these technologies over three years at Seattle Children’s.

Both Seattle Children’s and bluebird bio are working in the field of gene replacement therapies, where faulty genes causing inherited diseases are replaced by working healthy genes. Gene editing takes that idea one step further, repairing the faulty gene rather than replacing it. The researchers involved in the project believe gene editing may promote a more natural response to underlying genetic problems and offer a safer alternative to introducing a replacement gene from outside.

Support from bluebird will fund a five-person team at Seattle Children’s Center for Immunity and Immunotherapies researching gene editing applied to inherited blood diseases such as severe combined immunodeficiency, also known as bubble boy’s disease, and sickle cell disease. The gene editing tools studied are expected to focus on immunotherapies with gene editing applied to T-cells or blood-forming stem cells.

Under the agreement, Seattle Children’s scientists will get access to some of bluebird bio’s gene editing technologies. In June 2014, bluebird acquired Precision Genome Engineering, a developer of gene editing techniques with proteins the company says can recognize and cleave DNA more precisely and efficiently than other technologies. All of bluebird’s gene editing programs are still in preclinical stage.

The deal also gives bluebird bio development rights to future related discoveries. In addition, bluebird is licensing intellectual property based on research in Seattle Children’s labs. The nature of the licensed intellectual property was not disclosed.

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