Navigation Links
Amsterdam Molecular Therapeutics Reports Promising Data From Cholesterol Lowering Gene Therapy Study

AMSTERDAM, May 20, 2010 /PRNewswire-FirstCall/ --

- Gene Therapy Vector Successfully Delivers shRNA Into Liver Cells

Amsterdam Molecular Therapeutics (Euronext: AMT) a world leader in gene therapy, today reported positive preclinical data from a study using an AAV gene therapy product to lower cholesterol. The data show that a single dose of the gene therapy carrying a short hairpin RNA to silence Apolipoprotein B100 (ApoB100), resulted in a reduction of serum cholesterol of approximately 80%, without any signs of toxicity. These data validate AMT's technology platform as a powerful tool for successful gene silencing within target cells. The data were presented by Annemart Koorneef, scientist at AMT, yesterday during the 13th Annual American Society of Gene and Cell Therapy (ASGCT) Meeting in Washington, DC.

"With just a single dose of our cholesterol targeting gene therapy, a long-lasting, significant reduction of serum cholesterol is achieved. This preliminary study suggest that AMT's technology may have overcome one of the major problems of shRNA therapies, namely efficient and non-toxic intracellular delivery," noted Jorn Aldag, CEO of Amserdam Molecular Therapeutics.

In the study, AMT used its proprietary AAV-based platform to efficiently deliver a shRNA that silences both human and mouse Apolipoprotein B100 (ApoB100). A single intravenous administration caused prolonged ApoB100 gene silencing that was sequence-specific and not associated with liver toxicity, oversaturation of the cellular miRNA machinery or induction of immune responses. In addition, it was shown that the shRNA constructs against ApoB100 specifically and efficiently silence human ApoB100 ex vivo.

ApoB100 is the structural protein of Low Density Lipoprotein (LDL) particles that carry cholesterol. Silencing of ApoB100 with shRNAs results in a reduction of LDL-cholesterol and has the potential to be used to treat hypercholesterolaemia and cardiovascular disease.

About Amsterdam Molecular Therapeutics

AMT, founded in 1998 and based in Amsterdam, is a leader in the development of human gene based therapies. Using adeno-associated viral (AAV) 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 safe and efficacious proprietary platform offers a unique manufacturing capability which 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 LPL Deficiency, Hemophilia B, Duchenne Muscular Dystrophy, Acute Intermittent Porphyria and Parkinson's Disease at different stages of research or development.

AMT's lead product, Glybera, for the treatment of lipoprotein lipase deficiency has been filed for market authorization with the EMA. The current technology enables AMT to further expand its strategy to develop long-lasting therapies for severe metabolic diseases.

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 Amsterdam Molecular Therapeutics 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, including, but not limited to, the timely commencement and success of AMT's clinical trials and research endeavors, delays in receiving U.S. Food and Drug Administration or other regulatory approvals (i.e. EMEA, Health Canada), market acceptance of AMT's products, effectiveness of AMT's marketing and sales efforts, development of competing therapies and/or technologies, the terms of any future strategic alliances, the need for additional capital, the inability to obtain, or meet, conditions imposed for required governmental and regulatory approvals and consents. AMT expressly disclaims any intent or obligation to update these forward-looking statements except as required by law. For a more detailed description of the risk factors and uncertainties affecting AMT, refer to the prospectus of AMT's initial public offering on June 20, 2007, and AMT's public announcements made from time to time.

SOURCE Amsterdam Molecular Therapeutics B.V
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. BD Announces FDA 510(K) Submission of Rapid Molecular Test to Identify Patients Carrying Staph Superbug Prior to Surgery
2. Preclinical Data on Lead MEK Inhibitor, RDEA119, to be Presented at the 2007 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics
3. Access Pharmaceuticals to Present Data on ProLindac(TM) at the AACR-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference
4. Abbotts m2000(TM) Molecular Diagnostic Instrument and RealTime HIV-1 Test Win Chicago Innovation Award
5. Tapestry Pharmaceuticals Presents Data on TPI 287 at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics
6. Access Pharmaceuticals Announces Two Poster Presentations Reporting Significant Preclinical Data on ProLindac(TM) at the AACR-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference
7. Study Shows That Low-Molecular Weight Heparin (LMWH) was Associated with Reduced Hospital Costs of Venous Thromboembolism (VTE) Treatment Compared with Unfractionated Heparin (UFH)
8. Study of Pathwork Diagnostics Tissue of Origin Test Published in the Journal of Molecular Diagnostics: Investigates Test Reproducibility in Multiple Labs to Determine Primary Tumor in Cancer Cases
9. Beckman Coulter Announces Licensing Agreements for Molecular Tests with National University of Ireland, Galway
10. Molecular Biometrics Hires Denny Sakkas, PhD Renowned Yale Embryologist and International Thought-Leader, as Chief Scientific Officer; Opening U.S. Research Facility
11. Molecular Therapy Succeeds in Preventing Craniofacial Birth Defect in Mice
Post Your Comments:
(Date:11/30/2015)... 30, 2015 ... adds a 2015 publication on ... 2015 with comprehensive analysis of recent ... deal types, such as Mergers & ... . --> ...
(Date:11/30/2015)... 30, 2015  The fee-for-service reimbursement model ... medical imaging is on its way to ... payer-provider contracts are set to phase out ... alter provider-vendor relationships. The shift to value-based, ... new purchasing frameworks in the medical imaging ...
(Date:11/30/2015)... MOUNTLAKE TERRACE, Wash. and ST. ... Cross and Express Scripts (NASDAQ: ESRX ) today ... benefit agreement. The partnership, which began in 1999, will ... --> --> After evaluating pharmacy ... process, Premera concluded that Express Scripts continues to offer ...
Breaking Medicine Technology:
(Date:11/30/2015)... ... 01, 2015 , ... The National Association of Professional Women (NAPW) ... the Year Circle. She is recognized with this prestigious distinction for leadership in estate ... boasting more than 850,000 members and over 200 operating Local Chapters. , “I’m pleased ...
(Date:11/30/2015)... ... 30, 2015 , ... 1Heart Caregiver Services’ Executive Team actively ... Philippine American Chambers of Commerce held from November 6-8, 2015 at the Monte ... as an active delegate from the Philippine American Chamber of Commerce of Greater ...
(Date:11/30/2015)... ... November 30, 2015 , ... Being a caregiver for someone ... Maryland Health Care System, the Caregiver Support Program promotes the health and wellbeing ... job. Seventy-four percent report that their role as a caregiver has created marital ...
(Date:11/30/2015)... ... November 30, 2015 , ... During the National Family Caregivers ... held two webinars on topics of ‘Medical and Palliative Care Decisions,’ and ‘Self-Care ... . , With a loved one's diagnosis of mesothelioma, the closest family ...
(Date:11/30/2015)... ... November 30, 2015 , ... According to research by the ... dental technicians to be certified or obtain continuing education. To increase awareness of ... Your Mouth?” campaign to inform dentists that the technicians they trust could lack ...
Breaking Medicine News(10 mins):