Skip to main content
Premium Trial:

Request an Annual Quote

Amsterdam Molecular Therapeutics Says AAV-RNAi Approach Inhibits ApoB

Premium

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."

The Scan

Not Immediately Told

The US National Institutes of Health tells lawmakers that one of its grantees did not immediately report that it had developed a more infectious coronavirus, Science says.

Seems Effective in Kids

The Associated Press reports that the Pfizer-BioNTech SARS-CoV-2 vaccine for children appears to be highly effective at preventing symptomatic disease.

Intelligence Warning on Bioeconomy Threats

US intelligence warns over China's focus on technologies and data related to the bioeconomy, the New York Times reports.

PLOS Papers on Campylobacteriosis Sources, Inherited Retinal Dystrophies, Liver Cancer Prognosis

In PLOS this week: approach to uncover source of Campylobacteriosis, genetic risk factors for inherited retinal dystrophies, and more.