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AMT Accesses Technology for Treatment of Duchenne Muscular Dystrophy
Date:5/21/2008

AMSTERDAM, The Netherlands, May 22 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it obtained a license from La Sapienza University in Rome, Italy, to their advanced small nuclear RNA (snRNA)-based exon-skipping technology for the treatment of Duchenne muscular dystrophy (DMD). The combination with AMT's proprietary adeno-associated virus (AAV) gene therapy platform potentially makes up a long-term treatment for this seriously debilitating disease with a single administration of the product.

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy, caused by mutations in the dystrophin gene (essential for muscle function) is a severely debilitating neuromuscular disease. It affects young children, is progressive and leads to death in young adulthood. In the USA and Europe about 120,000 people suffer from it. Today, there is no treatment to prevent the fatal outcome.

The group of Prof. Irene Bozzoni at La Sapienza has done seminal work to develop a treatment for DMD. Using adeno-associated viral vectors, AMT's gene therapy platform of choice, and an exon-skipping snRNA program Bozzoni and co-workers demonstrated long term systemic and therapeutic effect in animals after a single administration[1]. One of the important advantages of the Bozzoni technology is that a single systemic delivery of the AAV vector containing the required antisense RNA-coding construct will result in long-term treatment of Duchenne muscular dystrophy.

Formalizing relationship with leading RNA research group of prof. Irene Bozzoni

Ronald Lorijn, CEO of AMT said: "We are very excited to have inlicenced an exciting therapeutic approach, which has shown to provide a life-long cure in rodents suffering from Duchenne's disease. In Duchenne's disease in particular the combined application of snRNA and AAV vectors has shown tremendous promise. Access to La Sapienza's
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SOURCE Amsterdam Molecular Therapeutics B.V
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