"Successful hepatocyte-specific delivery of microRNA (miRNA) and significant demonstration of gene silencing again illustrates the strength of AMT's AAV platform. We announced previously success with our short hairpin RNA targeting ApoB (shApoB) gene product but our newest data suggests our miRNA product is proving to be even better and safer," stated Jorn Aldag, CEO of AMT. "Certainly of the two approaches, the natural characteristics of miRNA, such as prolonged stable long term expression, undetectable toxicity profile, tissue specificity, as well as the significant efficacy, suggest a more favorable route to a viable treatment for high cholesterol. This future application of our technology may circumvent some of the delivery issues with the RNA approach. We intend to seek partners to exploit the full AAV technology potential in the RNA field."
Using its proprietary adeno-associated viral vectors (AAV), a single
injection of AAV-miApoB transduced murine hepatocytes almost entirely and
resulted in a reduction of total plasma cholesterol of 60-80% for an 18 week
period. AAV delivery of miRNA, expressed from a liver-specific promoter,
constitutes the second powerful approach by which AMT has demonstrated to
lower plasma cholesterol, together with AMT's AAV shApoB gene therapy product
tested in the same model of the disease. In our new approach, we went one
step further and limited the expression of the inhibitory miApoB molecule
only to the hepatocytes, which provides a higher safety profile, a feature
very important for future clinical applications. Data were presented at the
Hepatocyte User Group and Medicon Valley Hepatocyte User Forum (22-23
October) in Montpellier
|SOURCE Amsterdam Molecular Therapeutics B.V|
Copyright©2010 PR Newswire.
All rights reserved