Sickle Cell Disease: Remission of Signs of Disease in First Patient in World Treated with Gene Therapy; NEJM Publishes Case Study; Patient Treated with LentiGlobin Drug Product Is Free from Severe Symptoms & Has Resumed Normal Activities

On March 1, 2017. Bluebird Bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced publication in the New England Journal of Medicine of positive results of a case study on Patient 1204, the first patient with severe sickle cell disease (SCD) to be treated with gene therapy. The NEJM article is titled ““Gene Therapy in a Patient with Sickle Cell Disease.” This patient, who was 13 years old at the time of treatment, was treated with LentiGlobin drug product in the HGB-205 clinical study conducted in Necker Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France. The data in the publication reflects 15 months of follow-up. A brief summary of this patient’s outcomes with 21 months of follow-up was presented at the 58th American Society of Hematology Annual Meeting in December 2016. “We have managed this patient at Necker for more than 10 years, and standard treatments were not able to control his SCD symptoms. He had to receive blood transfusions every month to prevent severe pain crises,” said Professor Marina Cavazzana, M.D., Ph.D., principal investigator of this study and Professor of Hematology at Paris Descartes University, Head of the Department of Biotherapy Hospital, the clinical research center of Biotherapy at Necker Enfants Malades - Greater Paris University Hospital, AP-HP and INSERM, and of the Lymphohematopoiesis Laboratory, Imagine Institute of Genetic Diseases, Paris, France.
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