AVR-RD-02 Gene Therapy Still Showing Efficacy in 1st Treated Patient in Trial

AVR-RD-02 Gene Therapy Still Showing Efficacy in 1st Treated Patient in Trial

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AVR-RD-02 Gene Therapy Still Showing Efficacy in 1st Treated Patient in Trial

Updated, six-month data from a Phase 1/2 trial show a continued drop in the levels of two disease biomarkers in the first patient given a single infusion of AVR-RD-02, Avrobio’s experimental gene therapy for Gaucher disease type 1, the company announced. Results obtained by Jan. 4, the cutoff date marking six months post-treatment, also continued to show a positive safety profile, without serious or unexpected adverse side effects related to AVR-RD-02. The open-label trial (NCT04145037), called…

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