AMT Receives Innovation Credit from Dutch Government for Duchenne Muscular Dystrophy

07-Jan-2010 - Netherlands

Amsterdam Molecular Therapeutics announced that it will receive an Innovation Credit of up to € 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.

"We are delighted to receive this credit for DMD, as these awards are only made to innovative projects with strong commercial prospects," said Jörn Aldag, Chief Executive Officer of AMT. "This credit will allow us to prioritize the development of our gene therapy for this progressive and devastating disease."

Recently, AMT has reported it has successfully treated DMD in a preclinical model of the disease with its proprietary gene therapy product AMT-080. These proof of concept studies demonstrated that AMT’s technology resulted in functional dystrophin synthesis in both the heart and skeletal muscles, which prevents muscular dystrophy . These data reinforce a previous study in which this gene therapy approach was shown to successfully restore dystrophin in diseased human muscle cells obtained from biopsies of DMD patients. Together, these results establish a robust basis for AMT’s therapeutic approach to DMD.

The credit, together with accrued interest, will become repayable in mid-2013, subject to the commercial success of the project. The credit is payable in tranches linked to the achievement of specific milestones, and will fund 35% of the total anticipated costs of the project during this period.

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Topic world Gene therapy

Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.

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Topic world Gene therapy

Topic world Gene therapy

Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.