Oxford BioMedica
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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

UshStat® 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 October 2011, the FDA approved Oxford BioMedica’s IND application for UshStat® and in February 2012, the UshStat® Phase I/IIa study in Usher syndrome type 1B commenced in the US at the Oregon Health & Science University’s Casey Eye Institute. Led by Professor Richard Weleber as Principal Investigator, the open label, dose escalation Phase I/IIa study will enrol up to 18 patients and will evaluate three dose levels for safety, tolerability and aspects of biological activity. The Company is in the process of opening a second clinical site in France. Professor Jose-Alain Sahel will lead the French study at the Centre Hospitalier National D’Opthalmologie des Quinze-Vingts, Paris. On 29 June 2012, the Company announced that Sanofi had exercised its option to license UshStat® and in February 2014 the Company announced that the license agreement had been concluded.

Under the licence Sanofi were granted broadened global rights to UshStat® across all ocular indications and the Company is eligible for development and commercialisation milestone payments and royalties on any future sales. The parties expect that the management of the ongoing clinical studies will be handed over from the Company to Sanofi during the first half of 2014.

Market opportunity

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

Proof of concept

UshStat® 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 UshStat.
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


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