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Usher Syndrome Gene Therapy Begins

Usher Syndrome Gene Therapy Study Begins in Oregon

By Foundation Fighting Blindness (FFB, March 6, 2012)


The first-ever gene therapy for Usher syndrome, a devastating condition that causes both blindness and deafness, has moved into a Phase I/IIa clinical trial at the Foundation-funded Casey Eye Institute, Oregon Health & Science University (OHSU). The research team, led by Dr. Richard Weleber, is preparing to treat its first patient.

Developed by Oxford BioMedica, a biopharmaceutical company in the U.K. and a Foundation partner, the UshStat® treatment is designed to halt vision loss in people affected with Usher syndrome type 1B, which is caused by defects in the MY07A gene. Based on results in lab studies, researchers believe a single UshStat treatment may last several years, perhaps a lifetime.

“We are delighted to see Oxford’s innovative gene therapy for Usher 1B move into human studies. It is a critical milestone in our campaign to overcome vision loss from a particularly challenging condition,” says Dr. Stephen Rose, the Foundation’s chief research officer. “UshStat is great news for the Usher syndrome community, because there are virtually no vision-related treatment options available for any form of the disease. While the treatment is for Usher 1B, success in the trial will open the door for using gene therapy to treat other forms of Usher syndrome.”


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Thanks to Bob MacPherson