“The time is right for gene therapy. This funding will help us take the knowledge that we have acquired from 20 years of lab work to develop a clinical trial for the treatment of blindness.” – Dr. Catherine TsilfidisA team led by Dr. Catherine Tsilfidis has received $2.4 M from the Ontario Research Fund to develop treatments for retinal diseases, a leading cause of blindness. These diseases of the back of the eye are poorly understood, progressive and often untreatable. Dr. Tsilfidis’ research program aims to learn more about what causes them, and to create general therapies that can be applied to many kinds of eye disease. 

The first arm of the program will develop gene therapies that can prevent vision loss by keeping cells in the retina from dying. The gene therapy will use a protein called XIAP, which the team previously found could stop cell death in many forms of retinal disease in laboratory studies.

For example, their recently published study showed XIAP could significantly protect the optic nerve in a mouse model of   glaucoma. The XIAP gene will be delivered by injecting an adeno-associated virus (AAV) into the eye. AAVs have been shown to be relatively safe and effective for delivering gene therapy to the eyes. The virus will then transfer the gene to cells in the retina. 

The team will also complete the work required to bring XIAP gene therapy into clinical trials, which could happen in the next three to four years. The second arm of the program will investigate whether lost vision can be restored through stem cell transplants. The team will also test whether adding the XIAP protein to the mix can increase the long-term survival of these transplanted cells.

“The time is right for gene therapy. This funding will help us take the knowledge that we have acquired from 20 years of lab work to develop a clinical trial for the treatment of blindness.” – Dr. Catherine Tsilfidis, senior scientist at The Ottawa Hospital and associate professor at the University of Ottawa

Co-investigators:  Stuart Coupland, Brian Leonard, Michael Dolin, Bernard Hurley, Manoj Lalu, Dean Fergusson, Pierre Mattar, Valerie Wallace

Collaborators: John Bell, Fighting Blindness Canada, Neugen Pharma

Core Resources: Ottawa Methods Centre, Biotherapeutics Manufacturing Centre,  Blueprint Excelerator

The Ottawa Hospital is a leading academic health, research and learning hospital proudly affiliated with the University of Ottawa.   

Media Contact 
Amelia Buchanan
Senior Communication Specialist
Ottawa Hospital Research Institute
613-297-8315
ambuchanan@ohri.ca