Bluebird Bio Advances Experimental Drug for Genetic Brain Disorder

October 28, 2013

Research Test Tubes

Bluebird bio Inc., a Cambridge gene therapy company, announced that the first patient has undergone infusion with its Lenti-D drug product in a stem cell transplantation designed to slow the progression of a genetic brain disorder called childhood cerebral adrenoleukodystrophy (CCALD), reports. The patient is being treated at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.

“Boys with CCALD face significant risks of mortality and morbidity with allogeneic stem cell transplantation, the current standard of care treatment, if an optimally matched donor cannot be identified,” David Williams, MD, said in a statement. “Bluebird’s autologous Lenti-D drug product has the potential to circumvent this challenge and address an important unmet medical need for patients with this devastating disease.”

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