Trial of a Gene Therapy for Usher Syndrome to Begin
October 18, 2011 – A trial of a potential new gene therapy for one type of Usher syndrome has been approved in the United States and will begin shortly at the Casey Eye Institute in Portland, Oregon. This trial will evaluate a gene therapy called UshStat®, designed specifically for people with Usher syndrome type 1B.
This early trial will enrol up to 18 patients with Usher syndrome type 1B. The study, led by Professor Richard Weleber, will evaluate three dose levels for safety, tolerability and aspects of biological activity. It is expected to begin enrolling patients before the end of 2011.
Usher syndrome type 1B is one of the most common subtypes of Usher syndrome. Type 1B is caused by a mutation of the gene encoding myosin VIIA (MY07A). People with other types of Usher syndrome will not benefit from this therapy. Learn more about Usher syndrome and its sub-types.
UshStat® uses a specially designed virus, created by the company Oxford Biomedica, to deliver a corrected version of the MYO7A gene to the cells of the retina. On the basis of pre-clinical data, it is anticipated that a single application of UshStat® to the retina could provide long-term or potentially permanent stabilisation of vision.
“We are delighted to be partnering with Oxford BioMedica in the design and conduct of this; the first trial of gene replacement for retinitis pigmentosa associated with myosin 7A-deficient type I Usher syndrome,” says Dr. Richard Weleber, principal investigator for the study. “This trial represents a major milestone in the history of Usher syndrome. We conclude that the gene replacement therapy that will be evaluated in this trial has the potential to provide a substantial, durable benefit for the vision of these patients.”