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news.Oxford BioMedica has announced that its UshStat, a gene therapy for the treatment of usher syndrome 1B, has received orphan designation from the Committee for Orphan Medicinal Products of the European Medicines Agency.
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UshStat is designed to deliver a corrected version of the MYO7A gene into the cells of the retina, using the company’s LentiVector gene delivery technology. In collaboration with Sanofi-aventis, clinical development is expected to start in 2011.
John Dawson, chief executive officer of Oxford BioMedica, said: “We are delighted to have received orphan designation from the European Medicines Agency for another of our innovative LentiVector-based candidates targeting debilitating and progressive ocular diseases.
“This designation is an important step towards the start of clinical trials of UshStat for Usher syndrome 1B as part of our landmark collaboration with Sanofi-aventis to develop gene therapies in the field of ophthalmology.”