UshStat was developed using LentiVector platform technology, which is a gene delivery system, with applications in product development and discovery research.
The open label, dose escalation Phase I/IIa study will enrol around 18 patients with Usher syndrome type 1B at the Oregon Health and Science University’s Casey Eye Institute, Portland, Oregon.
The trial, which is likely to begin by the end of 2011, will assess three dose levels for safety, tolerability and aspects of biological activity.