AVR-RD-01 Gene Therapy Clears Fabry Patient of Toxic Substrate, Trial Reports

AVR-RD-01 Gene Therapy Clears Fabry Patient of Toxic Substrate, Trial Reports

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AVR-RD-01 Gene Therapy Clears Fabry Patient of Toxic Substrate, Trial Reports

The first patient dosed with AVR-RD-01, an investigational gene therapy being tested in the ongoing Phase 2 FAB-GT clinical trial in people with Fabry disease, showed a 100% reduction, or complete clearance of toxic substrate, in a kidney biopsy one year later.  This result, which meet the trial’s primary goal, was consistent with the first kidney biopsy from another patient, showing an 87% reduction in toxic substrate, as previously reported by Avrobio, the therapy’s developer.  These…

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