US biotech start-up bluebird bio (Nasdaq: BLUE) has presented new data from the ongoing HGB-205 and HGB-206 clinical studies evaluating its LentiGlobin BB305 product candidate in patients with beta-thalassemia major and severe sickle cell disease (SCD), at the 57th American Society of Hematology (ASH) Annual Meeting.
The data from the HGB-205 study were highlighted in an oral presentation by Marina Cavazzana, lead investigator of the HGB-205 study and professor of hematology at Paris Descartes University, head of the department of Biotherapy Hospital, the clinical research INSERM center of Biotherapy at Necker Enfants Malades, (Assistance Publique-Hôpitaux de Paris) and the Lymphohematopoiesis Laboratory, Institute of Genetic Diseases, Imagine, Paris, France. Data from the HGB-206 study were presented during a poster session by John Tisdale, senior investigator, molecular and clinical hematology branch at the National Institutes of Health.
“The data from the HGB-205 and HGB-206 studies further demonstrate the potential for gene therapy to make a meaningful and enduring difference in the lives of patients with beta-thalassemia major or severe SCD,” said David Davidson, chief medical officer, bluebird bio. “We are especially encouraged by the data from the HGB-205 study, which is demonstrating stable clinical benefit in patients with beta-thalassemia major and includes data from the first patient with severe SCD ever treated with gene therapy, now with a year of follow up. The data from the HGB-206 study are still very early, and we look forward to gaining greater clarity on the therapeutic profile of LentiGlobin in severe SCD as we treat more patients and obtain longer follow up in the coming year,” Dr Davidson noted.
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