bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, today reported financial results and operational highlights for the quarter ended March 31, 2014.
"The first quarter of 2014 has been very productive for bluebird bio," said Nick Leschly, chief bluebird. "We treated the first beta-thalassemia major subject in our U.S. based Northstar Study with LentiGlobin, opened an IND for our sickle cell disease study and we are now presenting preliminary beta-thalassemia major data at the upcoming European Hematology Association Congress. We look forward to sharing these early clinical data and thank all those involved in our LentiGlobin clinical trials for their commitment to finding a potential new treatment for patients with beta-thalassemia.”
First Quarter 2014 and Recent Business Highlights:
-- Announced that the first patient with beta-thalassemia major had been transplanted in the Northstar Study of our product candidate LentiGlobin-- Filed an investigational new drug application (IND) with the U.S. Food and Drug Administration for the clinical study of our LentiGlobin product candidate for the treatment of subjects with sickle cell disease. The IND for this study, HGB-206, is now active -- Our clinical abstract has been accepted for presentation at the 19 th European Hematology Association Congress to be held in Milan, Italy between June 13 and 16. We plan to present preliminary clinical results from our ongoing, phase 1/2 HGB-205 Study of our LentiGlobin product candidate for the treatment of beta-thalassemia major -- Filed an IND amendment to conduct the ALD-103 Study. The ALD-103 Study is an observational study of subjects with childhood cerebral adrenoleukodystrophy (CCALD) treated by allogeneic hematopoietic stem-cell transplant. This study, along with our phase 2/3 Starbeam Study evaluating our product candidate Lenti-D, is intended to provide us with additional information regarding the current standard of care for CCALD and the potential safety and efficacy advantages of Lenti-D