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Alnylam is developing ALN-PCS, an RNAi therapeutic for the treatment of hypercholesterolemia, or high levels of cholesterol in the blood. Hypercholesterolemia contributes to many diseases, most notably cardiovascular disease - the leading cause of death in the U.S.
ALN-PCS is a systemically delivered RNAi therapeutic targeting the gene proprotein convertase subtilisn/kexin type 9, or PCSK9, a genetically validated target involved in the metabolism of LDL cholesterol (LDLc), or "bad" cholesterol. Our RNAi therapeutic has the potential to lower tissue and circulating PCSK9 levels resulting in higher LDL receptor levels in the liver, and subsequently lower LDLc levels.
Pre-clinical data with ALN-PCS have shown specific silencing of PCSK9 mRNA and PCSK9 serum protein levels of up to 90%, with an ED50 (the dose that provides a 50% silencing effect) of approximately 0.06 mg/kg for both mRNA and protein reduction. These studies have also demonstrated a greater than 50% reduction in levels of LDLc, which is rapid and durable, lasting for weeks after a single dose.
In early 2013, The Medicines Company and Alnylam announced an exclusive global alliance to advance the ALN-PCS program. The collaboration includes program includes ALN-PCS02 an intravenously administered RNAi therapeutic which has completed a Phase I trial, and ALN-PCSsc a subcutaneously administered RNAi therapeutic currently in pre-clinical development. Alnylam will continue the program while funded by The Medicines Company for an estimated one to two years to complete certain pre-clinical and Phase I clinical studies. The Medicines Company will then lead and fund development from Phase II forward and commercialize the ALN-PCS program if successful.
For further information open: http://www.alnylam.com/Programs-and-Pipeline/Alnylam-5x15/Hypercholestoralemia.php