AMT's Cooperative Research and Development Agreement With NIH to Boost Productio... ( AMSTERDAM The Netherlands May 26 ...)

AMT's Cooperative Research and Development Agreement With NIH to Boost Production Capacity

Date:5/25/2008

AMSTERDAM, The Netherlands, May 26 /PRNewswire-FirstCall/ --

- Production Increase Brings Large Disease Areas within Reach

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it obtained a Cooperative Research and Development Agreement from the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH), Bethesda, Maryland, that promises to substantially enhance the power of AMT's gene therapy platform.

The Agreement

The Cooperative Research and Development Agreement (CRADA) with the NIH gives AMT the option to license the exclusive rights to the recombinant adeno-associated virus (rAAV) baculoviral manufacturing technology developed for treating Duchenne Muscular Dystrophy (DMD) by Robert Kotin, Ph.D., and colleagues at the NHLBI Laboratory of Biochemical Genetics. While AMT's current production platform is based on a 50-liter bioreactor set-up, the application of the NIH technology has been scaled-up to 250 liters, and allows for further expansion to 1,000 liters. These production levels are unique in the gene therapy field today and are relevant in the context of some of the products that AMT has under development that affect large patient populations, such as Factor IX for hemophilia B, IGF-I for liver cirrhosis, and AMT-011 for NASH. The scale-up is also important for treatment of Duchenne Muscular Dystrophy (DMD) for which AMT has just closed an agreement with La Sapienza University in Rome, Italy, since this indication requires a relatively high dose of gene therapy vector in order to correct the defect in all affected muscles.

Ronald Lorijn, CEO of AMT said: "This CRADA with the NIH provides AMT with a technology that in combination with our proprietary in-house platform adds a whole new dimension to our manufacturing platform. Our combined effort has the potential to greatly increase the therapeutic reach of our gene the
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AMT's Cooperative Research and Development Agreement With NIH to Boost Productio...( AMSTERDAM The Netherlands May 26 ...)