ESGCT 2022 – Sangamo strengthens its case in Fabry

This time last year, four gene therapies were vying for a piece of the Fabry disease market. Since then, one has dropped out, one has disappointed and the chances for another look unclear – leaving Sangamo as the leader in this race.

The group strengthened its hand today with updated results from its phase 1/2 Staar trial of ST-920, including long-awaited data on patients not receiving enzyme-replacement therapy (ERT). However, it is probably too early to make a call on ST-920’s durability. And there are other projects in development designed to improve convenience over the current standard of care which, if successful, could provide competition.