UshStat™

UshStat is a gene-based therapy for the treatment of Usher syndrome 1B. The disease is caused by a mutation of the gene encoding myosin VIIA (MY07A), which leads to progressive retinitis pigmentosa combined with a congenital hearing defect. UshStat uses the Company's LentiVector system to deliver a corrected version of the MYO7A gene. A single administration of the product could provide long-term or potentially permanent correction.

Preclinical development

UshStat has demonstrated efficacy in the only available preclinical model of Usher syndrome. Further preclinical optimisation studies are ongoing.

Sanofi-aventis collaboration

Oxford BioMedica entered a collaboration with sanofi-aventis in April 2009 to develop four LentiVector-based products for the treatment of ocular diseases, including UshStat. Oxford BioMedica is responsible for preclinical development and for conducting initial Phase I/II studies. Under the joint development plan, the companies aim to advance UshStat into Phase I/II development in 2011.

Market opportunity

Approximately 70,000 people in the USA and EU have Usher syndrome, which is the most prevalent form of deaf-blindness. Approximately 8,000 have Usher syndrome 1B. There are currently no treatments available for Usher syndrome 1B and UshStat has been granted orphan drug designation in the EU for this indication.