Bluebird bio is singing a sweet song today, to the tune of $60 million. That's the amount its lineup of investors--including the newly arrived Shire ($SHPGY)--is sinking into the Cambridge, MA-based biotech as it prepares a pivotal Phase II/III study for its lead gene therapy and brings along the next two programs in the pipeline. The new round brings the total funds raised to a whopping $110 million, a rare sum for drug developers of any stripe.
Bluebird's staff--which has swelled to about 40 and will soon add another 5 to 10 workers--has been making fast progress with its platform technology. The biotech aims at extracting autologous stem cells from patients with rare genetic diseases, modifying the cells with a gene therapy delivered by a viral vector, and injecting the modified cells back into the patients, which then are expected to migrate where they're needed to correct the disorder. At the top of its list of disease targets is childhood cerebral adrenoleukodystrophy (the lethal ALD), followed by beta-thalassemia and sickle cell disease.
The new money will be used to accomplish a variety of goals. But the most significant will be a pivotal study on some 12 to 15 ALD patients, which is likely to run about two years after launching in 2013, says CEO Nick Leschly. A second Phase I/II study in beta-thalassemia is also expected to get started next year.
"We do think the time is now for gene therapy," Leschly tells FierceBiotech. The fresh injection of funds arrives just after the first approval for a gene therapy, he adds. And it hasn't hurt that the company is producing human data on its programs at a time orphan diseases represent one of the hottest fields in the industry.
"It's been an interesting evolution," says the CEO. Maybe in the next year bluebird can do its first partnership, helping to spread the word about the broad list of conditions he believes the platform can address. For now, though, the emphasis remains on handling its own in-house programs with backing from some A-list investors.
Venture backers include: New investors Deerfield Partners, RA Capital, and Ramius Capital Group; two undisclosed "blue chip public investment funds" and existing investors Arch Venture Partners, Third Rock Ventures, TVM Capital and Forbion Capital Partners. Shire came in as a strategic investor.
- here's the press release