First-in-human universal gene therapy for blood disorder

Diamond-Blackfan anemia (DBA) is potentially life-threatening condition that can cause severe anemia and other abnormalities. It’s a rare genetic blood disorder that occurs when the bone marrow fails to make red blood cells. Currently the only cure is a stem cell transplant, but this is only a viable option for select patients. Otherwise, children with DBA require lifelong follow-up care to manage symptoms, such as steroids and blood transfusions.

New data publishing 11/11 in Cell Stem Cell provides strong support for the first-in-human universal gene therapy trial for DBA through regulated GATA1 expression. Notably, it’s one of the first examples of treating 30 different genetic mutations with a single vector. This clinical-grade vector is ready, and the team hopes to take it to clinical trial next.Vijay Sankaran, MD, PhD, lead and corresponding author of the paper, is available for comment if you’re interested in learning more.

Please reach out to Joelle Zaslow (joelle.zaslow@childrens.harvard.edu), Research and Science Communications Manager at Boston Children's Hospital, with any questions or media inquiries.