bluebird bio, Inc. (Nasdaq: BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, today announced that the first subject in its phase 2/3 childhood cerebral adrenoleukodystrophy (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,” stated David A. Williams, MD, Chief of hematology/oncology at Boston Children’s Hospital and Associate Chairman of pediatric oncology at Dana-Farber Cancer Institute. “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.”
“Successfully initiating treatment in the Starbeam study represents an important step towards improving outcomes for patients with CCALD and is a major milestone for bluebird and its lentiviral gene therapy platform,” stated Dave Davidson, MD, bluebird bio’s Chief Medical Officer.
About the Starbeam study (ALD-102)The phase 2/3 study is designed to evaluate the safety and efficacy of Lenti-D drug product in the treatment of subjects with childhood cerebral adrenoleukodystrophy, or CCALD, a rare, hereditary neurological disorder affecting young boys that is often fatal. The trial study is planned to enroll up to 15 boys who will be followed for 24 months following a transplant with bluebird bio’s lentiviral transduced stem cells, Lenti-D. During this 24 month period, patients will be assessed for the onset of major functional disabilities, and other key assessments of disease progression.