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ZyStor Therapeutics, Inc. Cleared to Begin Clinical Trial for Targeted Protein Therapeutic for the Treatment of Pompe Disease
Date:10/14/2009

ells in a variety of tissues. ZC-701 is a recombinant protein containing ZyStor's proprietary GILT tag fused to GAA, thereby allowing more efficient delivery of GAA to the lysosome of muscle cells. In preclinical research, ZC-701 was found to be both safe and highly efficacious in well-studied animal models. In animal models, preclinical efficacy was seen at doses much lower than those reported for the currently approved drug for the treatment of Pompe disease. It is anticipated that the first-in-human trial of ZC-701 will be conducted in late-onset Pompe patients.

Dr. Barry Byrne, Professor of Pediatrics and Molecular Genetics & Microbiology at the University of Florida College of Medicine, said, "The FDA approval of the ZyStor IND represents an important step in developing an improved treatment for Pompe patients. Clinical studies will show whether this new therapeutic approach provides the same benefit in humans as in preclinical testing."

About ZyStor Therapeutics, Inc.

ZyStor Therapeutics, Inc. is a privately held biotechnology company based in Milwaukee, Wisconsin developing a novel class of targeted protein therapeutics incorporating the Company's proprietary GILT technology that allows more efficient delivery of therapeutic enzymes to the lysosome and is thus applicable to the treatment of lysosomal storage diseases. This proprietary technology is termed Glycosylation Independent Lysosomal Targeting (GILT) because the peptide tag replaces mannose-6-phosphate (M6P) as the moiety normally targeting the lysosome. Use of a peptide targeting strategy not only improves lysosomal delivery of the therapeutic but also offers manufacturing advantages over conventional protein therapeutics for the treatment of LSDs.

This press release contains certain "forward-looking statements" and all forward-looking statements involve risks and uncertainty. Although the Company believes that the assumptions underlying
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SOURCE ZyStor Therapeutics, Inc.
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