This Year's Highlights

• Successful completion of proof-of-principle animal studies.
• Successful completion of GILT-tagged Pompe disease enzyme-replacement constructs.
• Successful completion of $8.5 million Series B financing.

Anticipated Highlights

• Completion of GILT-tagged Pompe disease therapeutic animal biodistribution and efficacy studies.
• Completion of GILT-tagged Fabry disease therapeutic animal biodistribution and efficacy studies.
• Completion of next round of funding with proceeds used to conduct GILT-tagged Pompe disease therapeutic Phase I/II clinical trial.

Corporate Mission

ZyStor Therapeutics, Inc. develops innovative therapeutics that will significantly improve the lives of patients who suffer from rare genetic disorders known as lysosomal storage diseases (LSDs). There are more than 40 known LSDs, each caused by a deficiency in a different lysosomal enzyme. ZyStor has developed a novel, proprietary technology, known as Glycosylation Independent Lysosomal Targeting (GILT), which improves the delivery of therapeutics that replace the missing lysosomal enzyme in LSD patients and has demonstrated in cell culture and in a well-characterized animal model that the GILT technology should provide several distinct advantages over other LSD therapeutics.

Based in Milwaukee, Wisconsin at the Innovation Center in the Milwaukee County Research Park, ZyStor completed an $8.5 million financing in September 2004. Mason Wells and Venture Investors led the syndicate of seven venture funds that participated in this financing. This syndicate also included Prolog Ventures, the State of Wisconsin Investment Board, Hexagon Investments, Apjohn Ventures and Stonehenge Capital. ZyStor expects to file an investigational new drug application (IND) for its lead LSD therapy for the treatment of Pompe Disease in 2007.