ZyStor's Glycosylation Independent Lysosomal Targeting (GILT) technology is the first peptide-based targeting technology that enables efficient targeting of enzyme replacement therapeutics to the lysosomal compartment of cells 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."

SOURCE ZyStor Therapeutics, Inc.