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Amsterdam Molecular Therapeutics Says AAV-RNAi Approach Inhibits ApoB

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Amsterdam Molecular Therapeutics this week announced preclinical data showing that its proprietary siRNA-based technology could be used to inhibit apolipoprotein B and trigger a drop in plasma cholesterol levels in mice.

The agent uses adeno-associated viral vectors to deliver apoB-targeting siRNA sequences that have been incorporated into microRNA scaffolds, according to the company. A single injection of the so-called AAV-miApoB into mice "transduced murine hepatocytes almost entirely and resulted in a reduction of total plasma cholesterol of 60 [to] 80 percent for an 18-week period," it added.

"Successful hepatocyte-specific delivery of microRNA and significant demonstration of gene silencing again illustrates the strength of [the firm's] AAV platform," AMT CEO Jorn Aldag said in a statement.

This "application of our technology may circumvent some of the delivery issues with the RNA approach," he added. "We intend to seek partners to exploit the full AAV technology potential in the RNA field."

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