ST-920 Gene Therapy Showing Safety, Signs of Efficacy in Trial

ST-920 Gene Therapy Showing Safety, Signs of Efficacy in Trial

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ST-920 Gene Therapy Showing Safety, Signs of Efficacy in Trial

Sangamo Therapeutics’ gene therapy candidate ST-920 safely and effectively increased and sustained the levels of the alpha-GalA protein, the enzyme that is lacking in people with Fabry disease, according to preliminary Phase 1/2 trial data. Four people have been treated at the first two therapy doses being evaluated — 5 trillion vector genomes per kg of body weight (vg/kg) and 10 trillion vg/kg. As of the Sept. 17 data cutoff date, all showed above normal alpha-GalA activity,…

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