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February 9, 2021

AVROBIO reports 100% kidney substrate reduction in Fabry disease trial

AVROBIO has reported a complete clearance of toxic substrate in the kidney biopsy of the first patient dosed with its plato gene therapy platform in the ongoing Phase II FAB-GT clinical trial of investigational ex vivo lentiviral gene therapy, AVR-RD-01, for Fabry disease. 

AVROBIO has reported a complete clearance of toxic substrate in the kidney biopsy of the first patient dosed with its plato gene therapy platform in the ongoing Phase II FAB-GT clinical trial of investigational ex vivo lentiviral gene therapy, AVR-RD-01, for Fabry disease.

Plato gene therapy platform has a lentiviral vector, a personalised conditioning regimen with precision dosing and an automated, closed manufacturing process for providing potent and consistent drug product from manufacturing sites globally at commercial scale.

The patient four is the first in the trial to be treated using the platform and a kidney biopsy in the Phase II trial showed a 100% reduction from an average of four globotriaosylceramide (Gb3) inclusions per peritubular capillary (PTC) at baseline to zero inclusions per PTC one year after dosing.

The primary efficacy endpoint of the trial is kidney substrate reduction and was used by the US Food and Drug Administration (FDA) to analyse and approve treatments for Fabry disease.

AVROBIO noted that two of four FAB-GT trial subjects had evaluable kidney biopsies. An 87% reduction, effective clearance of Gb3 inclusions per PTC compared to baseline was previously reported in patient one’s kidney biopsy.

So far, four patients were dosed in Phase II FAB-GT trial and five in the fully enrolled Phase I trial.

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Latest combined data across all cardiac measures for the FAB-GT patients demonstrate that they continue to show stable cardiac structure and function a year post-gene therapy.

AVROBIO also reported six-month data from Phase I/II trial of an investigational ex vivo lentiviral gene therapy, AVR-RD-02, for Gaucher disease type 1.

Data showed that plasma chitotriosidase levels decreased 49% and toxic metabolite lyso-Gb1 levels decreased 44% in the first patient at six months post-gene therapy versus pre-gene therapy ERT baseline levels.

Furthermore, all three cystinosis patients in the investigator-sponsored Phase I/II trial of AVR-RD-04 showed robust data across multiple measures and remain off cysteamine pills and eye drops, with trial enrolment completion expected this year.

AVROBIO president and CEO Geoff MacKay said: “With 13 patients dosed across three clinical programs, we have observed sustained and potentially transformative improvements in key biomarkers and functional metrics, with data from our Fabry disease program out three-and-a-half years after dosing.

“Additionally, enrollment activities for our Fabry disease trial are accelerating, giving us added confidence in our efforts to meet our goal of having dosed a cumulative 30 patients across all our clinical programs by the end of the year.”

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