Google
Trusted Resources: News & Events
Latest announcements and gatherings
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.
+myBinderRelated Content
-
The Teen Symposium: Engaging Adolescents and Young Adults With Sickle Cell Disease in Clinical Care and ResearchObjective: In the United States, sickle...
-
Phase 1 trial to test under-the-skin injection of sevuparin in sickle cell patientsModus Therapeutics is going to launch ...
-
bluebird bio Presents New Data from HGB-205 Study of LentiGlobinTM Drug Product in Patients with Transfusion-Depende...First patient with severe sickle cell di...
-
Investigational Oral Inhibitor IMR-687 Shows Promising Results in Ongoing Phase 2 Clinical TrialImara’s investigational oral inhibitor...
-
SCDAA Gene Therapy Town Hall – Webinarhttps://www.youtube.com/watch?v=J77Gq9yI...
-
Clinical trial offers hope to adults with sickle cell diseaseAt 33, Alexis Jones-Heggs has known a li...
-
Imara’s IMR-687 for SCD Receives FDA Rare Pediatric Disease DesignationThe U.S. Food and Drug Administration (F...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.