Nick Leschly - bluebird bio

Evangelizing for biotech's best case

Name: Nick Leschly
Title: CEO, bluebird bio

Since its emergence in 2010, bluebird bio ($BLUE) has strived to stand out in the biotech world, eschewing capital letters (and frustrating copy editors) with its name and, more weightily, betting big on gene therapy at a time when many in the industry remained wary of the field. And CEO Nick Leschly has long reflected the same irreverence, preferring on-brand blue sneakers over biopharma's requisite formal footwear and piloting the company from a private gamble to a Wall Street success story.

Leschly, billed as chief bluebird by the company, took the helm after he and his colleagues at Third Rock Ventures pitched in on its $35 million B round, moving forward with some proprietary technology that promised to succeed where other gene therapies failed. Bluebird's candidates target genetic disorders by using an engineered virus to harmlessly replace errant genes with corrective copies through a one-time infusion, potentially curing patients of debilitating disease. Or, as Leschly puts it, "super-cool, Buck Rogers kind of medicine."

The company's first target, beta-thalassemia major, results from a defective beta-globin gene that stops patients from producing the hemoglobin they need, often leading to symptoms like severe anemia and an enlarged spleen. Bluebird's in-development treatment, LentiGlobin BB305, has thus far freed patients from the chronic transfusions they used to need, leading the FDA to grant its coveted breakthrough-therapy designation and stirring hopes that the biotech may have found a functional cure.

And bluebird's ongoing clinical success has translated into a rising profile, as the company pulled off a $116 million IPO in 2013 and has seen its share value soar nearly 600% ever since. Beyond its work in beta-thalassemia major, bluebird is developing LentiGlobin BB305 for the more common sickle cell disease, working on a Phase II gene therapy for the rare childhood cerebral adrenoleukodystrophy, collaborating with Celgene ($CELG) in the hot CAR-T immunotherapy space and toiling in the nascent field of gene editing.

Through it all, Leschly has both presided over the company's rapid growth and served as its public face. And because of bluebird's high profile, he has become a perhaps unwitting evangelist for the promise of gene therapy. The field as a whole is in the midst of a second honeymoon among investors and researchers. Serious safety issues and deliverability woes long hampered R&D in the field, but, after years of work, a new generation of academics and investigators believes it has hammered out the right viral vectors to safely and predictably get the right genes to their target tissues, spurring renewed hope for widespread clinical success.

And bluebird is at the forefront of gene therapy 2.0, helping convince investors and investigators to buy back into the technology by focusing on the human side of the fight against inherited disease. Beta-thalassemia often amounts to a genetic death sentence for the children born with it, and, borrowing a skiing term, Leschly says his company is working under the guiding principle that "every child should have the right to have a bluebird day."

-- Damian Garde (email | Twitter)

For more:
Special Reports: FierceBiotech's 2012 Fierce 15 - bluebird bio | The top 10 biotech IPOs of 2013 - bluebird bio
Gene therapy pioneer bluebird bags a 'breakthrough' title for lead program
Bluebird posts more promising results for its rare disease therapy
Bluebird bio snares gene-editing tech in $156M buyout deal
Bluebird's gene therapy 2.0 weans beta-thalassemia patients off blood transfusions

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Nick Leschly - bluebird bio