SAR 422459 (Sanofi)

SAR 422459

Ophthalmology (Stargardt disease)
Phase II


Gene therapy for Stargardt disease

Licensed to Sanofi: Phase II ongoing

Stargardt disease

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.

Our approach

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.

Clinical status

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.

Market opportunity

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.