Tuesday, October 18, 2011

Phase I/IIa Gene Therapy trial for Usher Syndrome type 1B approved

Oxford BioMedica plc, the leading gene-based biopharmaceutical, has announced that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for the Phase I/IIa clinical development of UshStat®, a novel gene-based treatment for Usher syndrome type1B. UshStat® was designed and developed by Oxford BioMedica using the Company's proprietary LentiVector® platform technology and is the third programme to enter clinical development under the Phase I/II ocular collaboration agreement signed with Sanofi in April 2009.

The approval of the IND follows the decision by the US Recombinant DNA Advisory Committee (RAC) to approve the UshStat® Phase I/IIa protocol in May 2011. The open label, dose escalation Phase I/IIa study will enrol up to 18 patients with Usher syndrome type 1B at the Oregon Health and Science University’s Casey Eye Institute, Portland, Oregon, USA. The study, led by Professor Richard Weleber, will evaluate three dose levels for safety, tolerability and aspects of biological activity and is expected to be initiated by the end of 2011.
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 type1B. 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 theCompany's LentiVector® platform technology to deliver a corrected version of the MYO7A gene toaddress 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 type1B. UshStat® has received European and US Orphan Drug Designation which brings development, regulatory and commercial benefits.

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