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Oxford BioMedica gets FDA approval for Phase I/IIa trials of deaf/blindness drug - Proactiveinvestors (UK)
Oxford BioMedica (LON:OXB) announced this morning that the US Food and Drug Administration has given approval for its gene-based treatment for Usher Syndrome Type 1B to enter Phase I/IIa trials.
Usher Syndrome is the most common form of deaf-blindness and affects up to 50,000 patients in the US and Europe. One of its most comment forms is Usher Syndrome Type 1B, which leads to progressive retinitis pigmentosa combined with a congenital hearing defect and is caused by a mutation of the gene MY07A.
Oxford’s UshStat treatment uses the firm’s LentiVector platform technology to deliver a corrected version of the MY07A gene to address the vision loss associated with the disease. The firm said that, 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.
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 at Portland, Oregon.
“This is the third ocular [Investigational New Drug] approval that Oxford BioMedica has received from the US regulatory agencies over the last 12 months which represents an exceptional achievement for our R&D and regulatory teams,” said John Dawson, Oxford’s chief executive officer. “The continued progress of our ocular programmes partnered with Sanofi will further support the development path for other LentiVector platform products. With no approved treatment available for patients, we look forward to bringing UshStat into Phase I/IIa clinical development later this year.”
Oxford BioMedica (LON:OXB) announced this morning that the US Food and Drug Administration has given approval for its gene-based treatment for Usher Syndrome Type 1B to enter Phase I/IIa trials.
Usher Syndrome is the most common form of deaf-blindness and affects up to 50,000 patients in the US and Europe. One of its most comment forms is Usher Syndrome Type 1B, which leads to progressive retinitis pigmentosa combined with a congenital hearing defect and is caused by a mutation of the gene MY07A.
Oxford’s UshStat treatment uses the firm’s LentiVector platform technology to deliver a corrected version of the MY07A gene to address the vision loss associated with the disease. The firm said that, 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.
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 at Portland, Oregon.
“This is the third ocular [Investigational New Drug] approval that Oxford BioMedica has received from the US regulatory agencies over the last 12 months which represents an exceptional achievement for our R&D and regulatory teams,” said John Dawson, Oxford’s chief executive officer. “The continued progress of our ocular programmes partnered with Sanofi will further support the development path for other LentiVector platform products. With no approved treatment available for patients, we look forward to bringing UshStat into Phase I/IIa clinical development later this year.”