Gene therapy for Usher syndrome type 1B
Licensed to Sanofi: Phase I/IIa trial ongoing
Usher syndrome is the most common form of deaf-blindness, and affects approximately 30-50,000 people in the US and EU. Type 1B is one of the most prevalent subtypes, and is associated with a mutation of the gene encoding Myosin VIIA (MYO7A). This leads to progressive retinitis pigmentosa combined with a congenital hearing defect, resulting in deaf-blindness.
SAR421869 is designed to address retinitis pigmentosa and preserve patients’ vision. The gene therapy uses Oxford BioMedica’s LentiVector® technology to deliver a corrected version of the MYO7A gene to retinal cells with a single administration intended to provide potentially long-term or permanent stabilisation of ocular function.
Proof of concept
SAR421869 has demonstrated efficacy in the industry standard pre-clinical model of Usher syndrome.
SAR421869 is licensed to Sanofi, which is conducting a Phase I/IIa clinical study. Oxford BioMedica is entitled to development milestone payments and royalties on future sales.
There are currently no treatments for retinitis pigmentation associated with Usher syndrome type 1B and SAR421869 has received European and US Orphan Drug Designation. Oxford BioMedica estimates the potential market size for SAR421869 totals approximately $40 million.