Pre-Clinical Data with ALN-CC5 for Treatment of Complement-Mediated Diseases

Pre-Clinical Data with ALN-CC5 for Treatment of Complement-Mediated Diseases

We presented pre-clinical data with ALN-CC5, a subcutaneously administered RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases, at the 25th International Complement Workshop held September 14 – 18, 2014, in Rio de Janeiro, Brazil. Data demonstrated potent and clamped C5 knockdown as well as robust inhibition of complement activity in non-human primates for up to 100 days with a subcutaneous, monthly dosing regimen. Further, in a rat model of membranous nephropathy, ALN-CC5 administration resulted in a significant reduction in proteinuria due to complement-mediated disease activity in the kidney.



We believe that these results support a differentiated opportunity for RNAi-mediated C5 synthesis inhibition with ALN-CC5 – as compared with serum C5 blockade with monoclonal antibodies – in settings with high-level proteinuria.  ALN-CC5 could represent an attractive therapeutic strategy and potential new treatment option for patients with complement-mediated diseases, including membranous nephropathy.



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