Oxford BioMedica
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SAR 422459

A gene-based therapy for the treatment of Stargardt disease.

Licensed to Sanofi. Phase I/IIa trial ongoing

Stargardt disease

Stargardt disease is the most common juvenile degenerative retinal disease with a US and EU prevalence of approximately 80-100,000 patients (source: Walia et al. (2009), Macular Degeneration International). SAR 422459 is a gene-based therapy for the treatment of Stargardt disease. The disease is caused by mutation of the ABCR gene which leads to the degeneration of photoreceptors in the retina resulting in vision loss.

Our approach

SAR 422459 uses the Company’s LentiVector® platform technology 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. SAR 422459 aims to preserve vision by inserting healthy ABCR gene into retina.

Clinical status

Sanofi has recently disclosed that SAR422459 (for the treatment of Stargardt Disease), which was licensed from the Group in 2014, has entered Phase IIa development. The Group is entitled to future development milestones and royalties on this product.

Market opportunity

There are currently no approved treatments available for Stargardt disease and other potential strategies do not target the root cause of the disease. As such, SAR 422459 has received European and US Orphan Drug Designation which is expected to bring development, regulatory and commercial benefits. Oxford BioMedica estimates the market size for SAR 422459 to be around $500 million.

Proof of concept

SAR 422459 has demonstrated efficacy in the only available pre-clinical model of Stargardt disease. A single administration was effective for the duration of the six-month study.

Key publication:
Kong et al. Gene Therapy (2008) 15, 1311–1320: “Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy”

Transduction of photoreceptors with equine infectious anemia virus lentiviral vectors: safety and biodistribution of SAR 422459 for Stargardt disease.

Binley K, Widdowson P, Loader J, Kelleher M, Iqball S, Ferrige G, de Belin J, Carlucci M, Angell-Manning D, Hurst F, Ellis S, Miskin J, Fernandes A, Wong P, Allikmets R, Bergstrom C, Aaberg T, Yan J, Kong J, Gouras P, Prefontaine A, Vezina M, Bussieres M, Naylor S, Mitrophanous KA.

Invest Ophthalmol Vis Sci. 2013 Jun 12;54(6):4061-71. doi: 10.1167/iovs.13-11871.

Clinical trials

If you would like to find out more information about the status of the trials, please visit the clinicaltrials.gov website.

Facts

10-20yrs

Stargardt disease is sometimes called a juvenile macular dystrophy as it can first appear between the ages of 10 to 20 (source: RNIB)


Orphan Drug Designation

SAR 422459 has received European and US Orphan Drug Designation as there are no approved treatments for Stargardt disease

Vision: Stargart disease

Vision: Stargart disease

Illustration of visual field.
Courtesy: "Through Our Eyes", a gallery of personal visual perceptions contributed by the macular degeneration community, Macular Degeneration Support: www.mdsupport.org