Navigation Links
Amsterdam Molecular Therapeutics Successfully Lowers Cholesterol In Vivo With Enhanced Novel MicroRNA AAV Gene Therapy
Date:10/25/2010

Amsterdam Molecular Therapeutics Successfully Lowers Cholesterol In Vivo With Enhanced Novel MicroRNA AAV... -- AMSTERDAM, October 25, 2010 /PRNewswire-FirstCall/ --


Advanced Search Search

  
Close
  1. Send a release
  2. Member sign in
  3. Become a member
  4. For journalists
  5. Global sites
PR Newswire: news distribution, targeting and monitoring
  1. Products & Services
  2. Knowledge Center
  3. Browse News Releases
  4. Contact PR Newswire
Biotechnology, Health Care & Hospitals, Medical Pharmaceuticals, Clinical Trials & Medical Discoveries Click to view news release full screen  

Amsterdam Molecular Therapeutics Successfully Lowers Cholesterol In Vivo With Enhanced Novel MicroRNA AAV Gene Therapy

 

AMSTERDAM, October 25, 2010 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (AMT) Holding N.V. (Euronext: AMT), a leader in the development of gene based therapies, today announced that its gene therapy product incorporating siRNA sequences into microRNA scaffolds to silence Apolipoprotein B100 (AAV-miApoB) was able to significantly lower plasma cholesterol levels in vivo over a period of 18 weeks. These preliminary results suggest that this approach could lead to a treatment for high cholesterol in humans.

"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, France, and at the European Society for Gene and Cell Therapy Annual Conference (22-25 October) in Milan, Italy.

In the new study, mice received intra-venous injections with equal doses of 1011 gc per animal AAV-shApoB or AAV-miApoB and were examined for 18 weeks. Expression of the shApoB and miApoB resulted in 90% ApoB protein knock-down, associated with 80% cholesterol decrease in murine plasma for the first 6 weeks. However, after 8 weeks the effect of the shApoB started to wear off, while miApoB remained effective in ApoB and cholesterol reduction for up to 18 weeks. Ongoing research aims to determine the mechanism for the differences seen between long-term AAV-shApoB and AAV-miApoB efficacy in murine livers. We believe that the long-term stability of the miApoB is due to its lower toxicity and off-target properties compared to shApoB because expression of miApoB is specifically limited to hepatocytes.

ApoB100 is the structural protein of Low Density Lipoprotein (LDL) particles that carry cholesterol. Silencing the activity of ApoB100 with miRNA or shRNA lowers plasma LDL-cholesterol by 60-80% and has the potential to be used to treat hypercholesterolemia and associated cardiovascular disease.

About Amsterdam Molecular Therapeutics

AMT is a leader in the development of human gene based therapies. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. This proprietary platform can be applied to a large number of rare (orphan) diseases that are caused by one faulty gene. Currently, AMT has a product pipeline with several AAV-based gene therapy products in LPLD, Hemophilia B, Duchenne Muscular Dystrophy, Acute Intermittent Porphyria, and Parkinson's Disease at different stages of research or development. AMT was founded in 1998 and is based in Amsterdam.

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of AMT only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT's business. AMT expressly disclaims any intent or obligation to update any forward-looking statements herein except as required by law.


'/>"/>
SOURCE Amsterdam Molecular Therapeutics B.V
Copyright©2010 PR Newswire.
All rights reserved

Related biology technology :

1. Amsterdam Molecular Therapeutics Reports Half-Year Results 2010
2. Amsterdam Molecular Therapeutics Reports Full Year Results 2009
3. The University of Amsterdam Joins the MetaMiner Metabolic Diseases Project
4. Amsterdam Molecular Therapeutics Reports Half Year Results 2009
5. Amsterdam Molecular Therapeutics Reports Full Year Results 2008
6. Amsterdam Molecular Therapeutics Reports Half Year Results 2008
7. Heiner Dreismann, Ph.D., Former President of Roche Molecular Systems, Joins the HistoRx Board of Directors
8. Molecular Detections Detect-Ready™ MRSA Assay Demonstrates Strong Performance In Independent Comparative Study From Leading UK Hospital
9. QIAGEN and Abbott Enter Into Agreement on Molecular Tests for HIV, HCV and HPV
10. New Data Suggest Veracytes Molecular Test Improves Accuracy of Thyroid Nodule Diagnosis Using Fine Needle Aspirate (FNA) Samples
11. Launch of Two New Platforms for Microplate Analysis Expands Reader Portfolio and Ensures Molecular Devices Readers Meet all Combinations of Customer Needs
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/11/2016)... 11, 2016 --> ... "Company") (OTCQB: PSID), a life sciences company focused ... Thermomedics subsidiary, which markets the Caregiver® FDA-cleared non-contact ... in January 2016, including entering into agreements with ... sales growth, and establishing several near-term pipeline opportunities. ...
(Date:2/11/2016)... ... 11, 2016 , ... Reichert Technologies, which has created advanced ... the highest level of accuracy and quality with the addition of two new ... Refractometer. Accurate, reliable and tough enough for the most demanding applications, these ...
(Date:2/11/2016)... ... February 11, 2016 , ... Global Stem ... clinic in Quito, Ecuador. The new facility will provide advanced protocols and state-of-the-art ... around the world. , The new GSCG clinic is headed by four ...
(Date:2/10/2016)... February 10, 2016 Early-career researchers ... Peru , Uganda and ... in health and nutrition   Indonesia , ... and Yemen are being honored for ... They are also celebrated for mentoring young women scientists who are pursuing ...
Breaking Biology Technology:
(Date:2/3/2016)... 3, 2016 ... the "Emotion Detection and Recognition Market ... Others), Software Tools (Facial Expression, Voice Recognition ... Regions - Global forecast to 2020" ... http://www.researchandmarkets.com/research/d8zjcd/emotion_detection ) has announced the addition ...
(Date:2/2/2016)... YORK , Feb. 2, 2016 Technology Enhancements ... presents an analysis of the digital and computed radiography ... Malaysia , and Indonesia ... trends and market size, as well as regional market ... country and discusses market penetration and market attractiveness, both ...
(Date:2/1/2016)... Feb. 1, 2016  Today, the first day of ... plans to develop a first of its kind workplace ... IBM Watson. In the first application of ... IBM ), and Welltok will create a new ... with cognitive analytics, delivered on Welltok,s health optimization platform. ...
Breaking Biology News(10 mins):