Date: 2011-10-18
Type of information:
phase: 1-2a
Announcement:
Company: Oxford Biomedica (UK)
Product: UshStat®
Action mechanism: Usher syndrome 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.
Disease: Usher syndrome type 1B
Therapeutic area: Ophtalmology
Country: USA
Trial
details: 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. 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.
Latest
news: * Oxford BioMedica has announced that the 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 type 1B. 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.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. * This open label, dose escalation Phase I/IIa study has been initiated in February 2012. 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.
