Gene Therapy Clinical Trials Begin in Canada

Dr. Ian MacDonaldThe University of Alberta, in coordination with NightstaRx, have initiated a clinical trial for patients with Choroideremia using NightstaRx’s gene therapy product AAV2-REP1. The phase 1 trial is an open-label study designed to test the safety and preliminary efficacy of sub-retinal injection of AAV2-REP1. The trial will be performed at the University of Alberta under the supervision of Dr. Ian MacDdonald and will enroll 6 patients with identified CHM mutations. “For many of us, CHMers and researchers, this is an exciting time, one that has been anticipated for many years”, says MacDonald.
“The Choroideremia Research Foundation is thrilled at the initiation of clinical trials at the University of Alberta,” says Chris Moen, President of the CRF. “Dr. MacDonald has supported the CRF and Choroideremia patients for years, and we are excited to see his team, in conjunction with NightstaRx, bring gene therapy trials to Canada.This is a tremendous opportunity to make gene therapy accessible to patients in Canada who are losing their sight.”
Pre-clinical development of the AAV2-REP1 vector was performed by Profs. Miguel Seabra and Robert Maclaren with financial support from the Choroideremia Research Foundation. The vector is currently being studied by Prof. Maclaren in a Phase 1/2 clinical trial at the University of Oxford, UK. Results published in The Lancet Medical Journal in January 2014 reported that six months after treatment with this therapy, the first six patients showed improvement in their vision in dim light and the two patients who had impaired visual acuity at the start of the trial were able to read more lines on the eye chart. The Phase 1/2 study is currently ongoing and, according to the NightstaRx website, the company will soon announce its plans for future studies.
For more information on NighstaRx and its ongoing gene therapy work, please visit
To learn more about the University of Alberta clinical trial, you can visit the Choroideremia Gene Therapy at the University of Alberta website at or to the study page on the website.