Mar 26, 2019

“Gene Therapy Has Arrived!”

Canada’s first ocular gene therapy clinical trial was led by Dr. Ian MacDonald. The FBC was proud to be one of the funders who made this trial possible – as Dr. MacDonald said in a recent talk: “Gene therapy has arrived, and it has arrived in Canada.” We were thrilled that our partner in accessibility, AMI, was there to film Dr. MacDonald’s talk and interview him about the new era of gene therapy.

Dr. MacDonald’s talk was the keynote address at a recent Vision Quest education event. The event was focused on clinical trials and emerging therapies for patients living with inherited retinal diseases such as retinitis pigmentosa (RP), choroidermia, Usher syndrome, Leber congenital amaurosis (LCA), and other rare genetic eye diseases.

In addition to Dr. MacDonald’s talk, the event also featured a panel discussion with many of the lead clinicians who are involved with the FBC’s Patient Registry, including Dr. Elise Héon, who leads the site at SickKids, Dr. Johane Robitaille, who leads the site at IWK in Halifax, and Dr. Vaishnavi Batmanabane, who is the logistical lead for the registry at SickKids. The AMI special features key moments from this panel discussion where attendees were able to learn more about the Patient Registry and ask questions.

The event also featured talks from industry experts who are leading the development of many innovative treatments and overseeing the planning and implementation of clinical trials around the world. The fact that we were able to assemble this industry panel illustrates the tremendous progress that has happened in the area of inherited retinal diseases – the panel featured experts who were working on gene therapies, but also stem cell therapeutics, and new small molecule or “drug” approaches to treating these diseases that for a long time have been considered “untreatable.”

Dr. Peter Adamson, the Senior Vice President Ophthalmology for the company ProQR, shared exciting and very promising results from a clinical trial that is testing an experimental treatment for LCA. ProQR’s approach is not gene therapy, but it often gets confused with gene therapy because it is a form of personalized medicine that works only for patients diagnosed with LCA10 who have a specific mutation in the CEP290 gene. We described this RNA therapy approach in an earlier story, but we highly recommend that you learn it directly from Dr. Adamson!

This Vision Quest event was special to all of us at the FBC because it showcased the tremendous research progress that has happened and is happening today!

Thanks to the FBC supporters and to our Vision Quest sponsors for making these events such an impactful experience for everyone involved! Together, we are moving the research forward and helping to transform laboratory discoveries into life-changing treatments for the patients who need them.We hope to see you at one of our upcoming Vision Quest events in 2019!