FDA Approves Start of Usher Syndrome Gene Therapy Clinical Trial
October 18, 2011 - A clinical trial of UshStat®, a gene therapy for Usher syndrome type 1B, is on track to begin by the end of 2011, thanks to the FDA’s approval of the Investigative New Drug application submitted by Oxford BioMedica, the developer of the treatment. Oxford is in the process of working with the investigators to obtain the final approvals from local institutional review boards to initiate the study. When the trial proceeds it will be the first-ever human study of a vision treatment for Usher syndrome, the world’s leading cause of combined deafness and blindness, affecting as many as 50,000 people in the United States.
The Phase I/IIa clinical trial for UshStat® will be conducted at Casey Eye Institute, Oregon Health & Science University (OHSU), under the leadership of Dr. Richard Weleber, director of the Foundation-funded OHSU Research Center for the Study of Retinal Degenerative Diseases. As many as 18 patients will be enrolled in the study, which will evaluate three different doses of UshStat® for safety, tolerability, and biological activity. The Foundation Fighting Blindness funded the preclinical studies that made the UshStat® clinical trial possible.
“The FDA’s approval of the IND for UshStat® is a major milestone in the drive to overcome a particularly devastating disease,” says Dr. Stephen Rose, chief research officer of the Foundation. “For the many people struggling with Usher 1B, the treatment can’t come soon enough.”
Usher syndrome 1B is caused by defects in the gene MY07A, which plays multiple roles in the health and function of the retina. The UshStat® treatment delivers copies of the corrective MY07A gene to affected cells in the retina using Oxford’s LentiVector® technology. Based on a lentivirus, the LentiVector® technology involves a genetically engineered virus developed specifically for therapeutic purposes. Oxford’s gene delivery technology is currently the best available for delivering relatively large genes, such as MY07A. Based on preclinical studies, researchers believe a single application of UshStat® will last many years, perhaps a lifetime.
There are three primary types of Usher syndrome — types 1, 2 and 3 — and several subtypes represented by letters. Generally speaking, people with Usher syndrome type 1B are born with profound hearing loss and progressive vision loss that is diagnosed during childhood.
The planned UshStat® clinical trial will be Oxford’s third gene therapy clinical trial for a retinal degenerative disease. The company currently has a Phase I/IIa clinical trial of gene therapy for Stargardt disease (StarGen™) underway at OHSU and soon to open at the FFB-funded Center in Paris under the direction of Professor Jose Sahel, as well as a Phase I clinical study of gene therapy for wet age-related macular degeneration (RetinoStat®) taking place at Wilmer Eye Institute, Johns Hopkins Hospital. |
