Gene Therapy ARU-1801 Showing Early Efficacy, Safety in Phase 1/2 Trial

Gene Therapy ARU-1801 Showing Early Efficacy, Safety in Phase 1/2 Trial

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Gene Therapy ARU-1801 Showing Early Efficacy, Safety in Phase 1/2 Trial

ARU-1801, an experimental gene therapy for sickle cell disease (SCD), lowered the number of vaso-occlusive crises and led to  blood cells producing fetal hemoglobin in a first group of three treated patients, according to data from an ongoing Phase 1/2 trial. Favorable safety is also being seen, with two of these patients two years post-treatment at the time of this analysis and the third up to 10 months out. These findings from the MOMENTUM study (NCT02186418)…

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