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

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: Oxford BioMedica).  One of the most common subtypes is Usher syndrome 1B.  The disease is caused by a mutation of the gene encoding myosin VIIA (MY07A), which leads to progressive retinitis pigmentosa combined with a congenital hearing defect.

Our approach

UshStat® uses LentiVector® technology to deliver a healthy copy of the MYO7A gene directly to the retina. On the basis of non-clinical data, it is anticipated that a single administration of UshStat® could provide long-term or potentially permanent correction.  

Clinical status: in partnership with Sanofi

UshStat® is in Phase I/IIa development.  The ongoing open label, dose escalation Phase I/IIa study will enrol patients with Usher syndrome type 1B at the Oregon Health and Science University’s Casey Eye Institute, Portland, Oregon. The study, led by Professor Richard Weleber, will evaluate three dose levels for safety, tolerability and aspects of biological activity. A second clinical site at Centre Hospitalier National d’Ophtalmologie des Quinze-Vingts in Paris, France opened in 2013 with Professor José-Alain Sahel as Principal Investigator. Further information can be found by visiting http://clinicaltrials.gov/

In June 2012, Sanofi elected to exercise its options under the 2009 agreement to acquire exclusive worldwide licences for further development, manufacture and commercialisation of StarGen and UshStat®. Oxford BioMedica received the aggregate option exercise payments of US$3 million in July 2012 and is eligible for further development and commercialisation milestone payments and royalties on any future sales of the products.

Market opportunity

There are currently no treatments available for retinitis pigmentosa associated with Usher syndrome type 1B.  UshStat® has received European and US Orphan Drug Designation which brings development, regulatory and commercial benefits.

Pre-clinical 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”


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