Foundation Funding Usher Syndrome Gene Therapy Clinical Trial in Paris

July 23, 2012

The Foundation Fighting Blindness recently awarded $125,000 to the Centre Hospitalier National d’Ophtalmologie des Quinze-Vingts, a clinical trial site for UshStat®, a gene therapy developed by Oxford BioMedica for people with Usher syndrome type 1B (USH1B). The funding level is the amount needed for the site, located in Paris, France, to take part in the study.

The Phase I/II, 18-person study began at Oregon Health & Science University in early 2012. Researchers at the Paris site plan to begin treating participants by early 2013. The study is scheduled to run through December 2014. Evaluating safety is the primary goal of the trial, though investigators will also be looking at the treatment’s effect on the retina and vision.

Usher syndrome is the world’s leading cause of combined blindness and deafness. The condition affects approximately 45,000 people in the United States. USH1B is a form of the disease caused by defects in the gene MY07A. The UshStat treatment replaces copies of the defective MY07A gene in the retina with normal copies. Researchers believe a single treatment may halt the disease for several years, perhaps a lifetime. There are currently no therapies available for USH1B.

“We are very pleased to be supporting the UshStat trial in Paris,” says Dr. Stephen Rose, chief research officer, Foundation Fighting Blindness. “Led by Dr. José Sahel, the Paris team is one of the best retinal research groups in the world. They will do an outstanding job evaluating UshStat’s potential for saving vision in people affected by a particularly challenging condition.”

Oxford BioMedica is also conducting clinical trials of RetinoStat®, a gene therapy for wet age-related macular degeneration, and StarGen™, a gene therapy for Stargardt disease.

In addition to support for the UshStat human study, the Foundation funded critical laboratory studies that made all of Oxford BioMedica’s gene therapy clinical trials possible. Funds for the Paris site were provided, specifically, by the Foundation Fighting Blindness Clinical Research Institute, which is tasked with the role of overseeing and advancing promising treatments through human studies.

lab technician holding beaker