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Bluebird bio sings to tune of $9.3m as gene therapy shows promise

Authored by Karl Simpson

Bluebird Bio is rapidly gaining a very visible profile in the area of rare diseases. Backed by a stellar line-up of investors, including Mark Levin’s Third Rock Ventures, the company have just pocketed another $9.3m from the California Institute of Regenerative Medicine. This brings their investment levels to in excess of $138m from a range of different funding sources.

March 2010 – $35m in series B funding, including investors Third Rock Ventures and Genzyme Ventures

March 2011 – $4.2m – French Muscular Dystrophy Association

April 2011 – $30m – Series C – ARCH Venture Partners joins previous investors

July 2012 – $60m Series D – Shire Plc come in as strategic investor along with new investors Deerfield Partners, RA Capital, Ramius Capital Group join previous investors

October 2012 – $9.3m – California Institute for Regenerative Medicine

The company, led by Nick Leschly, who took the helm on an interim basis as part of the Third Rock Ventures – Entrepreneurs-In-Residence (EIR) programme but later converted to the full time CEO, keep stepping up and finding the funding necessary to drive their gene therapies for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell anemia. The company has also added to their board with Jeff Walsh and Daniel Lynch, former Chairman of Avila Therapeutics (acquired by Celgene) and Chairman of Michael Gillman’s Stromedix (acquired by Biogen IDEC). John Maraganore, former CEO of Alnylan Pharmaceuticals also joined the board.

Bluebird bio received their most recent funding from the California Institute for Regenerative Medicine’s Strategic Partnership Awards initiative for the study of LentGlobin, bluebird bio’s treatment of beta-thalassemia, a blood disorder that reduces production of the iron-containing protein, hemoglobin.

The Phase 1/2 study, which will look at the safety and efficacy of LentGlobin, will begin in the US in 2013, according to the company. A study is currently underway to study the feasibility, safety and efficacy of the treatment in sickle cell disease. Results reported in Nature showed stable expression of functional beta-globin resulting in transfusion independence for more than four years after a single treatment.

Having garnered serious backing from hallmark investors, plus having corporate venture investment from notable rare disease players Genzyme and Shire, a US and EU Orphan Drug designation for their gene therapy in adrenoleukodystrophy (ALD), plus a management team with serious kudos, Bluebird Bio is seriously singing a tune that the rest of market is listening to.

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