BCH-BB694 Gene Therapy Safely Treating Severe SCD Patients in Pilot Trial

BCH-BB694 Gene Therapy Safely Treating Severe SCD Patients in Pilot Trial

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BCH-BB694 Gene Therapy Safely Treating Severe SCD Patients in Pilot Trial

BCH-BB694 — an experimental gene therapy targeting the BCL11A gene — safely increased the levels of fetal hemoglobin and prevented disease-associated complications in six people with severe sickle cell disease (SCD), according to interim data from a Phase 1 clinical trial. These findings further support the feasibility and therapeutic value of approaches targeting BCL11A, which is involved in the suppression of fetal hemoglobin in adult red blood cells. Trial findings were reported in the study, “Post-Transcriptional…

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