Progress

In March of 2012 a medical research team at the University of Alberta headed by Dr. Ian MacDonald received a $1.3 million grant to complete a a clinical trial of a gene therapy treatment for choroideremia. The CRFC, along with the Foundation Fighting Blindness and the Canadian Institutes of Health Research, helped to fund this grant and make this trial a reality in Canada.

Dr. Jean Bennett at the University of Pennsylvania, Philadelphia has been awarded a Foundation Fighting Blindness Grant of $1.5 million USD to prepare pre-clinical application and begin a Phase 1 choroideremia gene therapy trial. Treatments are expected to being in the fall of 2012. Dr. Bennett’s work is funded in part by the Choroideremia Research Foundation, a sister organization to the CRFC.

In September 2011, researchers from Canada, the United States, Germany, France and the United Kingdom came together for the first ever International Choroideremia Symposium. Papers were presented on the clinical presentation of the disease, the development of animal models and current and future clinical trials aimed at curing and treating the disease.

In October 2011 the first choroideremia gene therapy trial began at Oxford University under the supervision of Dr. Robert MacLaren. This is a Phase I human clinical trial involving 12-15 subjects. One subject is treated per month. The gene therapy vector used was developed by Dr. Miguel Seabra at Imperial College, London, UK.