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AVROBIO Announces 100% Kidney Substrate Reduction at 12 Months Post-Gene Therapy in First Patient Dosed with plato Gene Therapy Platform in Fabry Disease Phase 2 Trial


Benzinga | Feb 8, 2021 07:07AM EST

AVROBIO Announces 100% Kidney Substrate Reduction at 12 Months Post-Gene Therapy in First Patient Dosed with plato Gene Therapy Platform in Fabry Disease Phase 2 Trial

Consistent with the 87% reduction in the first evaluable kidney biopsy, there is complete clearance of Gb3 inclusions in second evaluable kidney biopsy in Fabry disease Phase 2 trial

New Gaucher disease type 1 Phase 1/2 data show plasma chitotriosidase levels decreased 49% and toxic metabolite lyso-Gb1 levels decreased 44% in first patient at six months post-gene therapy, compared to patient's pre-gene therapy ERT baseline levels

Strong new cystinosis Phase 1/2 data across multiple measures for the three dosed patients, including substantial improvement in photophobia in first patient 12 months post-gene therapy

Investor and analyst presentation today at 8 a.m. ET






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