Patients and FamiliesStatement on Fabry Disease Program

Statement on Fabry Disease Program

We recently announced disappointing news about our 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 us to deprioritize our Fabry program. We intend to share relevant findings in the future.

We are committed to continuing to develop only the highest quality treatments for those living with rare diseases, and we continue to strive to create a world free of these genetic diseases. Even though we are no longer enrolling new participants into the FAB-GT clinical trial, we are preparing to follow participants in a 14-year long-term follow up study to continue to monitor safety and efficacy of the gene therapy.

We are grateful for all the participants and health care providers involved in our clinical trials and recognize the important contribution they have made to advancing the science of gene therapy. We share in their disappointment that the outcome was not what we would have hoped, and we are optimistic that the community maintains its dedication to research so other efforts underway can potentially find solutions to address unmet needs in Fabry disease.

Geoff MacKay
President & CEO