First Demonstration of Therapeutic Gene Silencing in Primates with Systemic RNAi

Alnylam Pharmaceuticals, Inc. announced the first published demonstration in primates that a systemically delivered RNAi therapeutic can potently silence an endogenous disease-causing gene in a clinically relevant manner. Alnylam and its collaborators at Protiva Biotherapeutics, Inc., demonstrated silencing in primates of the gene for apolipoprotein B (apoB), a protein involved in cholesterol metabolism, with clinically significant efficacy as demonstrated by reductions in levels of cholesterol and low-density lipoproteins (LDL). According to Alnylam, this peer-reviewed research, published in Nature, represents a major advance because it suggests that an RNAi therapeutic can be effective when delivered systemically using a dosage appropriate for application in future human clinical studies. "Source":[ http://www.bionity.com/news/e/53513/?pw=a&defop=and&wild=yes&sdate=01/01/1995&edate=03/31/2006].

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