Hello CFA Members, please see below this information on AVROBIO’s ongoing study (AVR-RD-01). Thank you!

AVROBIO, a leader in lentiviral-based gene therapies, is a clinical stage company developing disruptive therapies that have the potential to transform patients’ lives in a single dose.

Gene therapy involves the transplantation of normal genes into cells in order to correct genetic disorders. Gene therapy uses a vector to carry and deliver a working copy of a gene to the body’s cells. AVROBIO’s plato™ is designed to target underlying genetic disease by restoring normal gene function and enzyme production.


AVROBIO is conducting a study to evaluate the safety and efficacy of gene therapy for Fabry patients in Canada. Patients outside of Alberta may also apply. All travel will be reimbursed. For more information about AVROBIO please visit www.avrobio.com

Dr. Aneal Khan at the Children’s Alberta Hospital is heading this study.  If you or someone you know is a man with classic Fabry disease between the ages of 18 and 50 and has not been on treatment for Fabry disease over the last ten years please contact: aneal.khan@ahs.ca or Shelly Jelinski at Shelly.Jelinski@albertahealthservices.ca for more information. Please call Shelly at 403-955-7874