FFB Written Articles » Usher Syndrome
Print

Gene Therapy Clinical Trial Underway for Stargardt Disease

June 15, 2011 - The first-ever clinical trial of gene therapy for Stargardt disease, a juvenile form of macular degeneration, is now underway at Oregon Health & Science University (OHSU) in Portland.

Known as StarGen™, the treatment holds great promise for halting devastating vision loss for approximately 30,000 people in the United States and tens of thousands more around the world affected by the condition.

The treatment is being developed by Oxford Biomedica, a biopharmaceutical company in the United Kingdom. The Foundation Fighting Blindness funded many of the pivotal lab studies that made the StarGen clinical trial possible.

Most cases of Stargardt disease are caused by defects in the gene ABCA4. Oxford’s treatment involves injection of a manmade virus that delivers a healthy copy of ABCA4 into cells in the retina. Researchers believe a single treatment may be effective for several years or perhaps a lifetime.

Twenty-eight participants, ages 18 or older, will be enrolled in the Phase I/IIa study, which is primarily evaluating safety. For more information on the trial, visit www.FightBlindness.org/StarGen
or contact the study coordinator, Maureen Toomey, at (503) 494-3975.

StarGen will also be evaluated in a Phase I/IIa clinical trial at the
Centre Hospitalier Nationale D’Opthalmologie des Quinze-Vingts in Paris, France.
 

Back to top