Synageva BioPharma preclinical data highlighting ability of SBC-103 to reduce accumulation of substrate in brain of MPS III B animal model

Synageva BioPharma, a clinical stage biopharmaceutical company, has established preclinical data highlighting the ability of SBC-103 to reduce the accumulation of substrate in the brain of an MPS III B animal model. Accurate measurement of abnormal substrate in the brain has historically been more challenging in this disease and this is most likely a reflection of the methods used. Given the importance of being able to measure substrate to assess disease progression and the effects of enzyme replacement in lysosomal storage disease, Synageva has being working on developing improved methods for quantifying heparan sulfate disaccharides (HSD), the substrate that accumulates in MPS III B patients due to the NAGLU enzyme deficiency. This poster shows firstly that the method can clearly differentiate between affected and unaffected NAGLU deficient MPS III B mice and, secondly, that treatment with SBC-103, a recombinant human NAGLU, using intrathecal and intravenous dosing approaches produced dose-dependent HSD level reductions in the brain, liver and kidney tissues. These new data build on work presented last year in which Synageva demonstrated that it had been able to make for the first time recombinant human NAGLU enzyme with good mannose-6-phosphate dependent cellular uptake properties.

While Synageva’s MPS III B program is still in the preclinical stages of development, Synageva is aiming to initiate a natural history study in the second half of 2013, and is working toward initiating human clinical studies in 2014.

About Synageva BioPharma Corp.

Synageva is a clinical stage biopharmaceutical company focused on the discovery, development, and commercialization of therapeutic products for patients with life-threatening rare diseases and unmet medical need. Synageva has several protein therapeutics in its drug development pipeline. The company has a team with a proven record of bringing therapies to patients with rare diseases.

Further information regarding Synageva BioPharma Corp. is available at

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