Oxford BioMedica
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SAR 421869

A gene-based therapy for the treatment of Usher syndrome type 1B.

Licensed to Sanofi. Phase I / IIa trial ongoing

Usher syndrome

Usher syndrome is the most common form of deaf-blindness with a US and EU prevalence of approximately 30-50,000 patients (source: Boughman et al, 1983; Gandahl 1987; Hope et al, 1997; Spandau and Rohrschreider, 2002). One of the most common subtypes is Usher syndrome type 1B, which is associated with a mutation of the gene encoding Myosin VIIA (MYO7A). This leads to progressive retinitis pigmentosa combined with a congenital hearing defect.

Our approach

SAR 421869 aims to address the retinitis pigmentosa aspect of the disease and preserve vision using the Company’s LentiVector® platform technology to deliver a corrected version of the MYO7A gene to retinal cells. A single administration could provide long-term or potentially permanent stabilisation of ocular function.

Clinical status

In 2014, we announced that Sanofi had taken exclusive licenses to SAR422459 and SAR421869 for the treatment of Stargardt disease and Usher Syndrome type 1B respectively. As a result, Sanofi has taken over development and commercialisation activities, and we will receive milestone payments and royalties on future sales. Both lentiviral vector based products are in clinical development, and during 2015 we completed the technology transfer for clinical trial material production to Sanofi, which now controls the development of these two promising treatments.

Market opportunity

There are currently no treatments available for retinitis pigmentation associated with Usher syndrome type 1B. As such, SAR 421869 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 421869 to be $40 million.

Proof of concept

SAR 421869 has demonstrated efficacy in the only available pre-clinical model of Usher syndrome.

Key publication:
Hashimoto et al. Gene Therapy (2007), 1–11: “Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B”

EIAV-based retinal gene therapy in the shaker1 mouse model for usher syndrome type 1B: development of SAR 421869.
Zallocchi M, Binley K, Lad Y, Ellis S, Widdowson P, Iqball S, Scripps V, Kelleher M, Loader J, Miskin J, Peng YW, Wang WM, Cheung L, Delimont D, Mitrophanous KA, Cosgrove D.

PLoS One. 2014 Apr 4;9(4):e94272. doi: 10.1371/journal.pone.0094272. eCollection 2014

Clinical trials

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


IND approval

In October 2011, the US Food and Drug Administration (FDA) approved an Investigational New Drug (IND) application for the Phase I/IIa clinical development of UshStat®

Normal vision

Normal vision

A scene as it might be viewed by a person with normal vision.
Courtesy: National Eye Institute, National Institutes of Health (NEI/NIH) Ref#:EDS01

Vision: retinitis pigmentosa

Vision: retinitis pigmentosa

A scene as it might be viewed by a person with retinitis pigmentosa associated with Usher syndrome type 1B.
Courtesy: National Eye Institute, National Institutes of Health (NEI/NIH) Ref#:EDS07