AVROBIO Announces Clinical Data For Gaucher Disease Gene Therapy; Says 'Following positive feedback from FDA and MHRA, registrational, global Phase 2/3 clinical trial for GD3 planned for second half 2023'

New compelling clinical data from first-ever Gaucher disease type 3 (GD3) patient and four Gaucher disease type 1 (GD1) patients dosed with investigational AVR-RD-02

Data from first pediatric GD3 patient, the more severe, progressive form of Gaucher disease, show biochemical correction and improvement in major refractory element of disease 15 months post gene therapy

Data from first four adult patients dosed in GD1 clinical trial show important reductions below baseline ERT levels in liver and spleen volume up to two years post gene therapy

Following positive feedback from FDA and MHRA, registrational, global Phase 2/3 clinical trial for GD3 planned for second half 2023

AVROBIO believes plato® gene therapy platform is late-stage ready with no major CMC changes anticipated​