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Start of Phase I/II Gene Therapy Clinical Trial for Hemophilia B
Date:3/10/2010

after accidental trauma or medical interventions, because they do not have sufficient functioning of an essential blood clotting factor, called Factor IX.

AMT will build on the outcome of this exploratory trial and is preparing for additional clinical development to establish safety, tolerability and proof-of-concept with Factor IX gene therapy produced using AMT's proprietary, clinically validated production system. AMT has the exclusive commercialization rights to the Factor IX gene used in the St. Jude trial and has the ability to produce gene therapy product for hemophilia B at high quality on a commercial scale. Additional developmental work using AMT's production system is on-going at St. Jude with AMT support.

Jorn Aldag CEO of AMT said: "Dr. Andrew Davidoff and his group at St. Jude, together with Professor Nathwani in London, have done very important scientific work on hemophilia B. We are really looking forward to the results of the trial for continuing our collaboration, aiming for a real cure for patients with this bleeding disorder. Use of the Factor IX gene fits perfectly with AMT's proprietary gene therapy platform and our business strategy of developing cures for seriously debilitating orphan diseases. "

This hemophilia B gene therapy, administered
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SOURCE Amsterdam Molecular Therapeutics B.V
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