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Amsterdam Molecular Therapeutics Reports Promising Data From Cholesterol Lowering Gene Therapy Study
Date:5/20/2010

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 statemen
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SOURCE Amsterdam Molecular Therapeutics B.V
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