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ASH 2019 | BCL11A as therapeutic target in SCD: gene therapy trial

BCL11A regulates the fetal-adult hemoglobin switch; thus, its repression represents an appealing therapeutic target for sickle cell disease (SCD). David Williams, MD, Dana-Farber/Boston Children’s Cancer & Blood Disorders Center, Boston, MA, reports the results from an open-label, non-randomized, single center, pilot and feasibility study (NCT03282656). This gene therapy study evaluated the safety of a single infusion of autologous bone marrow-derived CD34+ cells transduced with a lentiviral vector containing a short hairpin RNA targeting BCL11A embedded in a microRNA scaffold (BCH-BB694; shmiR technology) in patients with SCD. Early results suggest an acceptable safety profile, validation of BCL11A as an effective target and mitigation of cellular pathology of SCD. This press briefing was recorded at the American Society of Hematology (ASH) 2019 Annual Meeting and Exposition in Orlando, FL.

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