ZyStor Therapeutics, Inc. is a privately-held biotechnology company developing enzyme replacement therapies with enhanced drug targeting for the treatment of lysosomal storage diseases (“LSDs”). LSDs are a group of about 50 rare genetic disorders in which the patient lacks one or more specific enzymes, resulting in an accumulation of undigested macromolecules inside the lysosomes of the body’s cells. The accumulation of these macromolecules causes lysosomes to enlarge in the cells of tissues and organs, eventually leading to cell degeneration that results in progressive deterioration in physical and/or mental state and can result in death. LSDs are extremely rare and fall within the FDA’s guidelines for orphan disease indications. In total, worldwide sales of enzyme replacement therapies for these diseases totaled approximately $2.8 billion in 2008. Despite the strong sales of these drugs, they are generally characterized by varying degrees of suboptimal ability to target the lysosome, need for the infusion of high doses and difficulty in manufacturing.

ZyStor’s lead product candidate, ZC-701, is an enzyme replacement therapy for Pompe disease that, based on pre-clinical evidence and manufacturing experience to date, overcomes these limitations. ZC-701, a recombinant chimeric human protein produced in a proprietary cell line, utilizes the Company’s patented Glycosylation Independent Lysosomal Targeting (“GILT”) technology. GILT combines a peptide tag with residues of human acid alpha-glucosidase (“GAA”), which is the enzyme that is deficient in Pompe disease patients. ZyStor believes that the binding properties of the GILT tag in ZC-701 enable significantly greater targeting to the lysosome with less systemic administration of the drug compared to Myozyme®, Genzyme Corporation’s enzyme replacement therapy for Pompe disease and the only product approved by the FDA for the indication.