UshStat utilizes the company’s LentiVector platform technology to deliver a corrected version of the MYO7A gene to address the vision loss associated with the disease.
The Phase I/IIa trial is an open label, dose escalation study which will enroll up to 18 patients with Usher syndrome type 1B at the Oregon Health and Science University’s Casey Eye Institute.
The pre-clinical data anticipated that a single application of UshStat to the retina could provide long-term or potentially permanent stabilisation of vision.
Oxford BioMedica CEO John Dawson said the continued progress of their ocular programmes partnered with Sanofi will further support the development path for other LentiVector platform products.
Casey Eye Institute principal investigator Richard Weleber said they conclude that the gene replacement therapy that will be evaluated in this trial has the potential to provide a substantial, durable benefit for the vision of these patients.