Oxford BioMedica plc, the leading gene-based biopharmaceutical company, has received the award of US$ 125,000 from the Foundation Fighting Blindness (FFB), a US non-profit organisation. The company receives award via its translational research arm, the Foundation Fighting Blindness Clinical Research Institute (FFBCRI) formerly known as the National Neurovision Research Institute (NNRI).

The FFB was an early funding collaborator of Oxford BioMedica’s pre-clinical ocular programmes and this award furthers the Company’s long-standing relationships with FFB and FFBCRI. The award will support the opening of a second clinical site in Paris, France for the ongoing UshStat phase I/IIa study, currently underway in the US.

UshStat is a novel gene-based treatment for Usher syndrome type 1B, designed and developed by Oxford BioMedica using its proprietary LentiVector platform technology, and is the third ocular product to enter clinical development under the Company's collaboration with Sanofi. In February 2012, Oxford BioMedica initiated an open label, dose escalation phase I/IIa study in the US led by Professor Richard Weleber at the Oregon Health & Science University’s Casey Eye Institute. The ongoing study will enroll up to 18 patients and will evaluate three dose levels for safety, tolerability and aspects of biological activity. Initial safety data from this study are expected in H2 2012.

Oxford BioMedica plans to open the second clinical site at Centre Hospitalier National d’Ophtalmologie des Quinze-Vingts in Paris, France with Professor José-Alain Sahel as Principal Investigator. Subject to receiving regulatory approval, the second clinical site is anticipated to open in early 2013, from which point patients could be treated in parallel at both sites.

Dr Stephen Rose, chief research officer at the Foundation Fighting Blindness, said: “We are very pleased to be supporting the UshStat trial in Paris. Professor José-Alain Sahel’s team is one of the best retinal research groups in the world. They will do an outstanding job evaluating UshStat’s potential for saving vision in people affected by a particularly challenging condition.”

Professor José-Alain Sahel of Centre Hospitalier National d’Ophtalmologie des Quinze-Vingts Paris, France commented: “We are very grateful to FFB for this support and are committed to testing this innovative approach with our partners, such as Pr Christine Petit (Pasteur Institute) who first identified disease-causing mutations in this gene. Our collaboration with Oxford BioMedica, the Portland team and FFB, already implemented with the ongoing StarGen™ trial, holds major promise for our patients.”

Stuart Naylor, Chief Scientific Officer of Oxford BioMedica, said: “Having worked with FFB since 2003, our strong relationship has underpinned the success we have had in bringing novel ocular gene therapies into clinical development. With no available treatments for patients with Usher syndrome type 1B, we are grateful for FFB’s support to boost this programme with an additional clinical site.”

Usher syndrome is the most common form of deaf-blindness which affects approximately 30,000-50,000 patients in the US and Europe. One of the most common subtypes is Usher syndrome type 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. UshStat uses the Company's LentiVector platform technology to deliver a corrected version of the MYO7A gene to address the vision loss associated with the disease. On the basis of pre-clinical data, it is anticipated that a single application of UshStat to the retina could provide long-term or potentially permanent stabilisation of vision. There are currently no approved treatments available for Usher syndrome type 1B. UshStat has received European and US Orphan Drug Designation which brings development, regulatory and commercial benefits.

Oxford BioMedica plc is a biopharmaceutical company developing innovative gene-based medicines and therapeutic vaccines that aim to improve the lives of patients with high unmet medical needs.