AVRO - Avrobio reports 100% kidney substrate reduction with plato gene therapy in Fabry disease shares up 20%

Avrobio (AVRO) jumps 20% premarket following an announcement of 100% reduction, or complete clearance, of toxic substrate in the kidney biopsy of the first patient dosed with the plato gene therapy platform in Phase 2 FAB-GT clinical trial of AVR-RD-01, an investigational gene therapy for Fabry disease.Kidney substrate reduction is the trial’s primary efficacy endpoint.The kidney biopsy for Patient 4 in the trial showed a reduction from an average of 4.0 globotriaosylceramide (Gb3) inclusions per peritubular capillary ((PTC)) at baseline to zero inclusions per PTC one year after dosing.The company also announced six-month data from the first patient dosed in the Phase 1/2 trial of AVR-RD-02, an investigational gene therapy for Gaucher disease type 1.The data show that 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 enzyme replacement therapy ((ERT)) baseline levels.Hemoglobin and platelet counts, which are typically

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Avrobio reports 100% kidney substrate reduction with plato gene therapy in Fabry disease, shares up 20%