Foundation News » Usher Syndrome

Usher Syndrome Gene Therapy Safe Thus Far In Three Patients

Nov. 20, 2012 – Oxford BioMedica, a leading gene therapy development company in the United Kingdom, has received a positive interim safety review of its ongoing Phase I/IIa clinical trial for the UshStat^® gene therapy for people with Usher syndrome type 1B. The first three patients experienced no adverse events for up to six months after treatment. The positive review by the study’s Data Safety Monitoring Board, an independent panel of ophthalmology and gene therapy experts, enables the company to treat additional patients at a higher dose level.

Oxford BioMedica also reported that its StarGen^™ gene therapy for Stargardt disease and RetinoStat^® gene therapy for wet age-related macular degeneration continue to perform well in human studies.

The Foundation Fighting Blindness funded critical laboratory research that made all three gene therapy clinical trials possible.

Eight patients — four with severe levels of disease and four with less severe levels — have experienced no serious adverse events for up to 16 months after treatment in the Phase I/IIa StarGen clinical trial. An additional four patients are now being treated with a higher dose.

In the Phase I RetinoStat clinical trial, nine patients have been treated at three dose levels, and an additional nine are now being treated at the target dose level. The therapy has been safe for up to 19 months. The company also reports that higher doses of RetinoStat are resulting in higher levels of the therapeutic protein in the patients’ eyes.

A major benefit of gene therapy is that a single treatment, administered through an ocular injection, may halt vision loss for several years or perhaps a lifetime.

Sanofi, an international pharmaceutical company, is partnering with Oxford BioMedica in the development of the StarGen and UshStat gene therapy products.