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Statement on NHLBI Decision to Pause the Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Sickle Cell Disease


bluebird bio, Inc. suspended its clinical trialexternal link exploring the curative potential of genetic therapy for sickle cell disease using its lentiviral vector (gene therapy delivery system), because two research participants in this trial developed myeloid neoplasms following gene therapy. Out of an abundance of caution, the National Heart, Lung, and Blood Institute (NHLBI), part of the NIH, has paused participant enrollment and gene transfers in an NHLBI-funded trial at the Boston Children’s Hospital, Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Sickle Cell Diseaseexternal link.

Since this trial began in February 2018, nine individuals with sickle cell disease have been treated. The Boston Children’s trial uses a different, yet related, vector, and targets a different gene than those used in the bluebird bio, Inc. trial. However, despite having no indications of such harm in the Boston Children’s trial, NHLBI is taking this action following the announcement that bluebird bio, Inc. temporarily suspended its study.

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