Cambridge, MA-based Bluebird Bio said today it raised $60 million in an oversubscribed Series D financing to further development and testing of its gene therapy platform to treat severe genetic diseases. The round included existing investors ARCH Venture Partners, Third Rock Ventures, TVM Capital, and Forbion Capital Partners, along with new investors Deerfield Partners, RA Capital, Ramius Capital Group, and two unnamed “public investment funds,” the company said in a statement. Irish drug giant Shire, which has been investing heavily in gene therapy and regenerative medicine, also joined up as a strategic investor.
Bluebird (which was originally named Genetix) raised $88 million in three previous rounds.
Bluebird CEO Nick Leschly says the funding will help advance the company’s two lead programs, which involve testing its platform in the diseases childhood cerebral adrenoleukodystrophy (ALD) and beta-thalassemia. Leschly adds that the cash influx will also allow Bluebird to pursue a gene therapy treatment for sickle cell disease. “We’ve attracted a mature set of investors who are thinking about Bluebird in the context of a 10- or 15-year time horizon,” Leschly says. “It’s important that they’re willing to make a long-term bet.”
Indeed, Bluebird is attempting to break new ground in the controversial field of gene therapy. The company’s approach involves taking stem cells from the patient’s bone marrow, then inserting a healthy version of the disease-causing gene into it. The cells are then grown in culture and put back to the patient in a one-time procedure.
Bluebird has tested the therapy in four children with ALD, which is the progressive neurological disease that was portrayed in the film Lorenzo’s Oil. Two of the trial participants are still doing well five years post-treatment, Leschly says. The company plans to start a trial in 12 to 18 patients next year, he says, which he expects will be the pivotal trial required for FDA and European approval. “For two or three years subsequent to treatment we’ll be looking at how the brain is doing, and at what we call the ‘functional score,’ which is at the end of the day what parents care about. Are we able to stabilize the disease?”
On June 19, Bluebird announced that the FDA and the European Medicines Agency granted orphan status to the company’s ALD treatment, which guarantees it several years of market exclusivity if it is approved.
Bluebird’s other major program is in beta-thalassemia, a blood disorder characterized by a shortage of oxygen-carrying hemoglobin. Patients with the disease have to get blood transfusions on a regular basis. Bluebird’s gene therapy approach is designed to restart the body’s own hemoglobin production, and in the first patient that tried the therapy, it did just that. The patient no longer needs blood transfusions, Leschly reports. A second patient was treated in November, and the company plans to treat seven more before moving into a late-stage clinical trial.
For the next set of patients, the company plans to transition to a new “viral vector”—the construct it uses to transport the healthy genes into cells.”We’ve made dramatic improvements over the last two years in our ability to make it in a productive and potent way,” Leschly says.
The company is also examining whether the same treatment will work for sickle cell disease, a far more prevalent disorder that could represent a major market opportunity, Leschly says. “This funding allows us, as we kick off 2013, to contemplate a completely independent development plan for sickle cell disease,” Leschly says.
Leschly says the new funding will take Bluebird well into 2015, but he expects the company will need to secure some development partners to help finance late-stage development and commercialization. Bluebird’s pharma investors could be the key to securing those partners, Leschly says. Sanofi unit Genzyme invested in the company in 2010. “Adding Shire to the mix is great,” he says, noting Shire’s market leadership in rare diseases. “With their knowledge of the orphan space, it will be helpful to have them at the table.”