March 22, 2011 7:04 AM
by John Carroll
Bluebird bio's gene therapy hits a high note
Over the last week, Cambridge, MA-based bluebird bio has taken several key steps forward. The gene therapy pioneer named Alexandre LeBeaut as its new chief medical officer, signed up an impressive roster of experts for its scientific advisory board and inked a $4.2 million pact with the French Muscular Dystrophy Association to develop a treatment for beta-thalassemia and sickle cell anemia.
For bluebird bio CEO Nick Leschly, the series of moves help represent a turning point for a biotech determined to prove that its novel approach can make a big difference in the treatment of rare genetic conditions--an increasingly popular field in biopharma in which rare diseases represent significant market opportunities.
"I think, broadly speaking, this is a really, really exciting time," Leschly tells FierceBiotech Research. The staff is likely to swell to up to the 30 to 35 range (from 25 to 30) in the near future and there's enough money in the bank--from the likes of Third Rock Ventures, TVM Capital, Forbion Capital Partners, Easton Capital and Genzyme Ventures--to get through 2012 and possibly into early 2013. During that time, bluebird plans to demonstrate the potential of its approach: Taking stem cells from a patient's bone marrow, adding a healthy version of a disease-causing gene and then growing it in culture before returning the treatment back to the patient.
Bluebird's LentiGlobin inserts a fully functional human beta-globin gene into the patient's hematopoietic stem cells. Researchers are conducting a Phase I/II trial examining the safety and efficacy of LentiGlobin in the treatment of beta-thalassemia and sickle cell anemia. And the platform technology can be used to design treatments for other genetic targets as well.
"We never use the word cure," says Leschly, who's quick to add that the developer is still some years away from any possible NDA. "This is such a delicate population; we're cautious about overpromising and under-developing." But if bluebird bio is right, they are on track to advance a potentially transformative approach that skirts some obvious potential complications associated with cell transplantation.
- check out the release on bluebird's new CMO
- read the release on the new scientific advisory board
- see the announcement on the deal with the French Muscular Dystrophy Association
Related Articles:
Bluebird in spotlight after gene therapy cures blood disorder
Cincinnati team taking sickle cell cure into the clinic
--Ankur
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