Gene therapy for Stargardt disease
Licensed to Sanofi: Phase II ongoing
Stargardt disease is the most common degenerative retinal disease in young people, and affects approximately 80-100,000 in the US and EU. The disease is caused by mutation of the ABCR gene, which leads to the degeneration of photoreceptors in the retina causing vision loss.
SAR422459 uses Oxford Biomedica’s LentiVector® technology to deliver a corrected ABCR gene via a single administration directly to the retina, which offers a potentially long-term or permanent correction.
Proof of concept
SAR422459 has demonstrated efficacy in the industry standard pre-clinical model of Stargardt disease, in which a single administration was effective for the whole of the six-month study.
SAR422459 is licensed to Sanofi, which has progressed the product into Phase II development. Oxford Biomedica is entitled to future development milestones and royalty payments on SAR422459.
There are no approved treatments available for Stargardt disease, and other therapeutic approaches do not target its root cause. Consequently, SAR422459 has received European and US Orphan Drug Designation. Oxford Biomedica estimates the market opportunity for SAR422459 totals approximately $500 million.