Approach involves engineering patients’ hematopoietic cells for treating beta thalassemia, sickle cell anemia, and CCALD.

Bluebird bio (formerly Genetix Pharmaceuticals) raised $30 million through a financing round with new investor Arch Venture Partners and existing investors. The firm says the funds will provide it with the financial stability to continue developing its clinical gene-therapy pipeline and invest in new product development and platform capabilities.

Just last month Bluebird was awarded an initial $1.4 million from the French Muscular Dystrophy Association (AFM) to support clinical development of its LentiGlobin® candidate for beta-thalassemia and sickle cell anemia. As part of the AFM deal Bluebird also has the option to draw on an additional amount of up to $2.8 million in credit toward the cost of cGMP manufacturing of clinical trials material by its manufacturing partner Généthon.  

Bluebird is focused on the development of gene therapies for severe genetic disorders. The company’s technology platform is based on the genetic correction of bone marrow stem cells harvested from the patient, and then reimplantation of the modified cells back into the patient.  The platform is being applied to single gene diseases that are currently treatable by allogeneic stem cell transplantation. Bluebird says that correcting the patient’s own cells could feasibly enable treatment of the 80% or more of patients who don’t have a matched related donor.

The firm has two clinical-stage stage products in development: for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell anemia. Its lead LentiGlobin® therapy is currently in Phase I/II trials for the treatment of beta thalassemia and sickle cell anemia. The approach involves engineering the patient’s own hematopoietic stem cells to express a fully functional human beta-globin gene, under the control of the beta-globin promoter and locus control regions. Results from a single LentiGlobin-treated beta-thalassemic patient in the Phase I/II trial were published in September last year. The young adult patient had been transfusion-dependent since early childhood, but after therapy with LentiGlobin became transfusion-independent for at least 21 months.

Bluebird’s CCALD gene therapy has undergone initial pilot testing. Data from a small-scale trial confirmed that two CCALD patients treated using the firm’s gene-therapy had stable disease at three years, and continued to express the CCALD protein in a percentage of monocytes. The firm says it plans to start larger clinical studies in CCALD in both the U.S. and Europe during 2011.

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