In an announcement today, Oxford BioMedica said that it had gained approval from the FDA to begin a Phase I/IIa Clinical Trial for a form of Usher’s Syndrome, Type 1B, which leads to progressive retinitis pigmentosa combined with a congenital hearing defect. Usher syndrome is the most common form of deaf-blindness which affects approximately 30,000-50,000 patients in the U.S. and Europe. One of the most common subtypes is Usher syndrome type 1B. The disease is caused by a mutation of the gene encoding myosin VIIA (MY07A). The open label, dose escalation Phase I/IIa study will enrol up to 18 patients with Usher syndrome type 1B at the Oregon Health and Science University's Casey Eye Institute , Portland, Oregon. The study, led by Professor Richard Weleber, will evaluate three dose levels for safety, tolerability and aspects of biological activity and is expected to be initiated by the end of 2011. Here is the complete news release from Oxford BioMedica: Oxford BioMedica Announces U...
Gene Therapy in Ophthalmology Update 13 New Clinical Site for Usher Syndrome Clinical Trials
In joint news releases, both the Foudation Fighting Blindness (FFB) and Oxford BioMedica announced that FFB had recently awarded $125,000 to the Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, a clinical trial site for UshStat, a gene therapy developed by Oxford BioMedica for people with Usher syndrome type 1B (USH1B). The funding level is the amount needed for the site, located in Paris, France, to take part in the study. Usher syndrome is the world's leading cause of combined blindness and deafness. The condition affects approximately 45,000 people in the United States. USH1B is a form of the disease caused by defects in the gene MY07A. The UshStat treatment replaces copies of the defective MY07A gene in the retina with normal copies. Researchers believe a single treatment may halt the disease for several years, perhaps a lifetime. There are currently no therapies available for USH1B. UshStat is a novel gene-based treatment for Usher syndrome type 1B, designe...