Avro­bio touts ear­ly clin­i­cal da­ta and plans new gene ther­a­py tri­al in 2023

Those with Gauch­er dis­ease have to prac­ti­cal­ly live at the hos­pi­tal to re­ceive bi-week­ly, hours-long in­fu­sions of en­zyme re­place­ment ther­a­py — for the rest of their lives. It’s in­con­ve­nient and can be cost­ly.

Avro­bio, a clin­i­cal-stage gene ther­a­py com­pa­ny, is try­ing to change how Gauch­er is treat­ed by de­vel­op­ing a one-time gene ther­a­py called AVR-RD-02 that has shown promis­ing, al­beit very ear­ly, re­sults for Gauch­er dis­ease Types I and III in two sep­a­rate tri­als.

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