Avrobio recently announced disappointing news about their Phase 2 FAB-GT gene therapy clinical development program for Fabry disease, which showed variable cell engraftment, a key indicator of efficacy, among the five most recently dosed patients. It’s important to note that this was not a safety issue. Unfortunately, these new data, as well as an increasingly challenging market and regulatory environment for Fabry disease, have caused them to deprioritize their Fabry program. They are working to better understand these data and intend to publish any relevant findings in the future.
Read the letter to the Fabry Community here