According to the UK based company Oxford Biomedica (LSE: OXB), the US FDA has given its approval for the initiation of an open label dose escalation Phase I/IIa clinical trial for “UshStat” a gene based treatment for Usher’s Syndrome Type 1B. The UshStat Phase I/IIa protocol was previously approved by the US Recombinant DNA Advisory Committee (RAC) in May. The common form of deaf-blindness affects between 30,000 and 50,000 patients in Europe and the US and is caused by a mutation in the myosin VIIA (MYO7A) gene. To date, there are no therapies or treatments available. The new experimental approach will aim to deliver a corrected version of the myosin VIIA gene in a lentiviral vector to the sub-retinal space. The study, expected to begin before the end of 2011 under the direction of Professor Richard Weleber, will initially recruit up to 18 patients with the disease at the Oregon Health & Science University’s Casey Eye Institute in Portland, Oregon.
UK company reports FDA approval of Phase I/II study for Usher’s syndrome type 1B
- by swdadmin