On October 8, 2009 AMT received Orphan Drug Designation for its treatment for Duchenne Muscular Dystrophy (DMD).
The Company remains on track to file for regulatory approval of its lead product Glybera(R) within the next three months. This gene therapy is to control LPLD, a serious disease often complicated by potentially life threatening pancreatitis incidents.
AMT will continue to develop its own technology platform and exploit its advantages in AAV gene therapy by focusing its preclinical development on 4 projects: Hemophilia B, Duchenne Muscular Dystrophy (DMD), Acute Intermittent Porphyria (AIP) and Parkinson's Disease. Other projects previously shown in the Company's pipeline will be postponed or discontinued to control the Company's cash burn.
About Amsterdam Molecular Therapeutics
AMT has a unique gene therapy platform that appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. 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. As such, AMT's proprietary platform holds tremendous promise for thousands of rare (orphan) diseases, especially diseases that are caused by one faulty gene. Currently, AMT has a pipeline with several AAV-based gene therapy products at different stages of research or development.
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," "go
|SOURCE Amsterdam Molecular Therapeutics B.V|
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