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AMT Successful in Preclinical Treatment of DMD

Wednesday, November 11, 2009 General News
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AMSTERDAM, November 11 Amsterdam MolecularTherapeutics (Euronext: AMT), a leader in the field of human gene therapy,announced today that it has successfully treated Duchenne muscular dystrophy(DMD) in an animal model with its proprietary gene therapy. The proof ofconcept studies were performed in collaboration with the group of ProfessorIrene Bozzoni (University of Rome, La Sapienza, Italy) and demonstratedeffectiveness in the heart as well as in skeletal muscles. In a previousstudy, AMT's gene therapy approach was shown to be successful in thetreatment of diseased human muscle cells obtained from biopsies of DMDpatients. These data establish a robust basis for AMT's therapeutic approachto DMD.
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"We are proud to establish proof of concept with our gene therapy forDuchenne muscular dystrophy, as it is an important new step in developing atreatment for this progressive and devastating disease," said Jörn Aldag,Chief Executive Officer of AMT. "In particular, our positive results in theheart and respiratory muscles bring hope that we might be able to prevent thefatal outcome of this disease in the future, potentially through a long-termremedy with a single treatment."
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About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a severe disease characterized byprogressive muscle degeneration. It affects young children, almostexclusively boys, and leads to paralysis and death in young adulthood. Thedisease is caused by mutations in the dystrophin gene, as a result of whichthe production of functional dystrophin protein, an important structuralcomponent within muscle tissue, is blocked. Currently, there is no treatmentto prevent the fatal outcome of this disease. DMD affects one in 3,500 males,making it the most prevalent of muscular dystrophies.

AMT is developing a gene therapy product for DMD based on 'exon skipping'technology which results in bypassing the genetic defect such that thefunctional protein can be formed again. Positive long-term therapeuticeffects of this approach have been demonstrated in animals.

About Amsterdam Molecular Therapeutics

AMT has a unique gene therapy platform that appears to circumvent many ifnot all of the obstacles that have prevented gene therapy from becoming amainstay of clinical medicine. Using adeno-associated viral (AAV) vectors asthe delivery vehicle of choice for therapeutic genes, the company has beenable to design and validate what is probably the first stable and scalableAAV production platform. As such, AMT's proprietary platform holds tremendouspromise for thousands of rare (orphan) diseases, especially diseases that arecaused by one faulty gene. Currently, AMT has a pipeline with nine productsat 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 futureoperations, prospects and financial condition. Words such as "strategy,""expects," "plans," "anticipates," "believes," "will," "continues,""estimates," "intends," "projects," "goals," "targets" and other words ofsimilar meaning are intended to identify such forward-looking statements.Such statements are based on the current expectations of the management ofAmsterdam Molecular Therapeutics only. Undue reliance should not be placed onthese statements because, by their nature, they are subject to known andunknown risks and can be affected by factors that are beyond the control ofAMT. Actual results could differ materially from current expectations due toa number of factors and uncertainties affecting AMT's business, including,but not limited to, the timely commencement and success of AMT's clinicaltrials and research endeavors, delays in receiving U.S. Food and DrugAdministration or other regulatory approvals (i.e. EMEA, Health Canada),market acceptance of AMT's products, effectiveness of AMT's marketing andsales efforts, development of competing therapies and/or technologies, theterms of any future strategic alliances, the need for additional capital, theinability to obtain, or meet, conditions imposed for required governmentaland regulatory approvals and consents. AMT expressly disclaims any intent orobligation to update these forward-looking statements except as required bylaw. For a more detailed description of the risk factors and uncertaintiesaffecting AMT, refer to the prospectus of AMT's initial public offering onJune 20, 2007, and AMT's public announcements made from time to time.

SOURCE Amsterdam Molecular Therapeutics B.V
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