Bluebird bio Inc., a Cambridge gene therapy company that had an intial public stock offering in June, said Monday the first patient has has undergone infusion with bluebird bio’s Lenti-D drug product in a stem cell transplantation.
The company, which looks to develop next generation products to treat patients with severe genetic and orphan diseases, has been working on a therapy to slow the progression of a genetic brain disorder called childhood cerebral adrenoleukodystrophy, or CCALD.
In a Monday press release, bluebird bio announced that the first subject in its phase 2/3 CCALD study, Starbeam (ALD-102), has undergone infusion with bluebird bio’s Lenti-D drug product in an autologous hematopoietic stem cell transplantation.
“Treating our first subject in this study reflects the recent advances in the field of gene therapy and is the culmination of years of collaborative effort between the team at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and our colleagues at Massachusetts General Hospital, INSERM in Paris, and bluebird bio,” David A. Williams, MD, chief of hematology/oncology at Boston Children’s Hospital and associate chairman of pediatric oncology at Dana-Farber Cancer Institute, said in a statement. “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. 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.”
Bluebird bio is one of more than half a dozen Massachusetts biotech companies that have gone public so far this year, including Enanta Pharmaceuticals Inc. and Tetraphase Pharmaceuticals Inc. of Watertown and Cambridge-based Epizyme Inc., Agios Pharmaceuticals Inc., and Acceleron Pharma Inc.