AMT has shown efficacy in studies of a preclinical model of DMD. These proof of concept studies demonstrated that AMT's technology resulted in functional dystrophin synthesis in both the heart and skeletal muscles, leading to the prevention of muscular dystrophy. These data are strengthened by a study in which this gene therapy approach was shown to successfully restore dystrophin activity in diseased human muscle cells obtained from biopsies of DMD patients. A Phase I/II clinical trial is scheduled to start by the end of 2012.
AMT has received an Innovation Credit of up to EUR 4 million from the Dutch government to support the development of AMT's gene therapy treatment for Duchenne Muscular Dystrophy (DMD). The credit is granted by SenterNovem, an agency of the Dutch Ministry of Economic affairs.
"It is exceptional that we have been able to reveal the promise of this therapy to the FDA in this early stage of the development. We believe our proven adenoassociated viral vector technology used in all our gene therapy products provides a distinct advantage. AMT has successfully conducted three clinical trials with its lead product Glybera that employs this technology, confirming that AAV-based delivery technology is safe and efficacious," noted JÃ¶rn Aldag, CEO of Amsterdam Molecular Therapeutics.
The FDA's orphan drug designation is intended to encourage research and
development of new therapies for diseases that affect fewer than 200,000 U.S.
residents. As a designated orphan drug, AMT-080 is eligible for tax credits
based on its clinical development costs, as well as assistance from the FDA
in guiding the drug through the regulatory
|SOURCE Amsterdam Molecular Therapeutics B.V|
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