Platform: Single mutation gene therapy
Company: bluebird bio
Therapeutic opportunities: Single-gene disorders
Gene therapy has been around for more than 20 years--the first trial was in 1990--but it has never reached the great potential originally envisioned and has run into a host of clinical problems. Today, some companies continue betting on gene therapy, but with a second-generation twist.
Cambridge, MA-based bluebird bio is addressing a different gene therapy need: It's going after single-gene mutation diseases that have few or no clinical options, including childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell anemia.
Bluebird takes stem cells from a patient's bone marrow, introduces a healthy version of the gene via a lentiviral vector, and grows it in culture before reinfusing the corrected stem cells back to the patient. This process could eliminate the need for, and potential problems associated with, donor cell transplantation, such as engraftment failure and the risk of graft-versus-host disease.
"In the next two years, bluebird looks to have its ALD program well into a Phase II/III trial and two other programs nearing completion of Phase I/II trials for beta-thalassemia and sickle cell disease," the company's chief medical officer, David Davidson, said in February.
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 the company attracted $60 million in VC support in July in part to fund the key study.
The company had a significant success in 2010 when one of its beta-thalassemia patients was able to stop receiving monthly blood transfusions for two years after his gene therapy treatment. The results were published in the journal Nature.
(With reports from FierceBiotech Editor-in-chief John Carroll)