University of Alberta Begins First Gene Therapy Trial for Choroideremia in Canada

Monday, August 3 2015 | 00 h 00 min | Vision Science

The University of Alberta has recently begun the enrolling and dosing of subjects in a Phase II clinical trial for a gene therapy treatment of choroideremia, a rare inherited disorder where the capillaries of the choroid degenerate, causing blindness by around middle age.

This gene therapy, in which a viral vector carrying a working copy of the REP1 gene is introduced via sub-retinal injection, was developed by NightstaRx, a UK-based biopharmaceutical company, specializing in the development of therapies for retinal dystrophies. The current phase of this trial involves six subjects, all of them men.

Ian MacDonald, Professor of Ophthalmology and Visual Sciences at the University of Alberta and leader of the clinical research team, says, “It’s a great privilege to be able to do something very positive for people with choroideremia. People have hoped for this for a long time.”

Further Information: http://www.albertahealthservices.ca/11502.asp