Bluebird Bio – 2012 Fierce 15

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Nick Leschly, CEO of Bluebird Bio

Based: Cambridge, MA
Founded: 2009
CEO: Nick Leschly
Clinical Focus: Gene therapy
Website: bluebirdbio.com

The Scoop: Nick Leschly finds himself running a company that's positioned at the crossroads of a resurgent gene therapy technology and the hot development space for new drugs that treat rare diseases. Just weeks ago, the biotech announced that it had won orphan status in the U.S. and Europe for its lead program, a gene therapy that can treat adrenoleukodystrophy, or ALD.

In a tiny study, quite common for rare diseases like ALD, Bluebird delivered early-stage proof-of-concept evidence that their lentiviral delivery tech could deliver a corrective gene straight to a patient's stem cells, a one-time therapy spurring the kind of proliferation needed to deliver the treatment to the brain. Two of four patients have benefited from stable protein expression for four years. And the technology has other applications in other diseases, creating a potential set of research partnerships for the company as it prepares a pivotal Phase II/Phase III study.

What Makes it Fierce?

Bluebird has enlisted a loyal group of top-line venture groups. And it needs them. The company's studies may not require many patients, but they're expensive. Over four venture rounds, Bluebird, previously named Genetix, has raised a whopping $110 million, which will get the biotech into 2015, with a chance to earn some partnership cash along the way.

Leschly--who joined the company in 2010 when it switched to the Bluebird name--knows venture financing. He was part of the Millennium Pharmaceuticals team that went on to form Third Rock back in 2007. And he's played a hand in several of the venture group's startups, including Agios and Edimer.

Next year, Bluebird plans to launch a pivotal study with 12 to 18 ALD patients, targeting a neurological affliction that was the subject of the movie "Lorenzo's Oil." And there's more to come.

The neurodegenerative disorder CCALD, beta-thalassemia and sickle cell disease are all on Bluebird's radar. In one patient with beta-thalassemia, a blood disorder, Bluebird's approach restarted hemoglobin production, eliminating the need for blood transfusions. And it's attracted some serious attention from potential partners.

"We're talking to some big companies about areas we can't go into on our own," says the CEO. And if Bluebird can replicate its success seen among a handful of patients, it's likely to win even more attention from Big Pharma companies interested in developing the market for high-cost rare disease treatments.

Investors: Deerfield Partners, RA Capital, and Ramius Capital Group; two undisclosed "blue chip public investment funds," Arch Venture Partners, Third Rock Ventures, TVM Capital and Forbion Capital Partners. Shire came in as a strategic investor. Another rare disease player, Genzyme Ventures, contributed cash in 2010.

-- John Carroll (Twitter | LinkedIn)