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

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

We have presented pre-clinical data with ALN-CC5, an RNAi therapeutic for the treatment of complement-mediated diseases, at the 6th International Conference on Complement Therapeutics being held June 18 – 23, 2013. The data showed that administration of a GalNAc-siRNA conjugate targeting the C5 component of complement resulted in potent, dose-dependent, and durable silencing of C5 liver mRNA, knockdown of C5 serum protein levels, and inhibition of complement-mediated hemolytic activity in rodents.



 

We are excited by the potential for an RNAi therapeutic targeting C5 for the treatment of complement-mediated diseases, as it possesses several characteristics that comprise our “Alnylam 5×15” product development strategy. Specifically, C5 is predominantly expressed in the liver and is a genetically and clinically validated target. Furthermore, our clinical development plan will be facilitated by serum biomarkers in Phase I trials and, if successful, a relatively streamlined and focused path for advanced development. We believe that a subcutaneously administered RNAi therapeutic targeting C5 could represent a very promising strategy for the treatment of a broad range of complement-mediated diseases.



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