Gene Therapy Trial for Retinitis Pigmentosa Based on Usher’s Syndrome To Begin
October 25, 2011
Oxford BioMedica announced that it had gained approval from the FDA to begin a Phase I/IIa Clinical Trial for a form of Usher’s Syndrome, Type 1B, which leads to progressive retinitis pigmentosa combined with a congenital hearing defect.
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).
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.
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