One-time AVR-RD-01 Gene Therapy Safe Up to 4.5 Years, Trials Report

One-time AVR-RD-01 Gene Therapy Safe Up to 4.5 Years, Trials Report

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One-time AVR-RD-01 Gene Therapy Safe Up to 4.5 Years, Trials Report

A single dose of Avrobio’s investigational gene therapy AVR-RD-01 continues to show a favorable safety and tolerability profile for up to 4.5 years in adolescents and adults with Fabry disease, according to latest data from Phase 1 and Phase 2 clinical trials. Notably, up to 3.5-year data previously showed the therapy was also effective at increasing the levels of alpha-galactosidase A (Gal A), the faulty enzyme in Fabry patients, and in reducing the levels of disease biomarkers. Updated…

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