StarGen™

StarGen is a gene-based therapy for the treatment of Stargardt disease. The disease is caused by a mutation of the ABCR gene which leads to the degeneration of photoreceptors in the retina and vision loss. StarGen uses the Company's LentiVector system to deliver a corrected version of the ABCR gene. A single administration of the product directly to the retina could provide long-term or potentially permanent correction.

Preclinical development

StarGen has demonstrated efficacy in the only available preclinical model of Stargardt disease. A single administration was effective for the duration of the six-month study. The preclinical development is being supported by the Foundation Fighting Blindness, through its translational research arm, the National Neurovision Research Institute.

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 StarGen. 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 StarGen into Phase I/II development in 2010.

Market opportunity

Stargardt disease is the most common juvenile degenerative retinal disease with a US and EU prevalence of approximately 50,000 patients and 600 new cases per year. There are currently no treatments available for Stargardt disease and StarGen has been granted orphan drug designation in the EU for this indication. Other potential opportunities are cone-rod dystrophy and the dry form of age-related macular degeneration, where the same mutant gene plays a role. These indications would significantly expand the market opportunity for StarGen.