ZyStor receives first funding and buys theraputics for rare diseases

Milwaukee — ZyStor Theraputics, Inc., has received an undisclosed piece of its $8.5 million financing and bought technology from St. Louis-based Symbiontics, Inc., for treating rare diseases, the company said.

The investment from a syndicate of seven venture funds drew Symbiontics executives to Wisconsin, forming a new company, ZyStor, to buy its technology.

• Read WTN’s report on ZyStor’s formation and the Wisconsin investment that caught its attention.

ZyStor expects to file an investigational new drug application in 2006 with the FDA for a treatment for lysosomal storage diseases, which hinder enzymes in the body. These enzymes normally break down proteins, sugars and fats. Without them, the build-up can harm the skeleton, lungs, heart, and other organs. It is sometimes fatal.

There are more than 40 such diseases, each causing a deficiency in a particular enzyme.

Company officials previously said they would first target Pompe, Fabry’s and Gaucher’s diseases. Each affects 10,000 or fewer people worldwide. Nevertheless, Genzyme, the market leader, makes $800 million a year on its treatment for Gaucher’s disease. One patient’s treatment for a year runs $160,000.

The technology ZyStor bought from Symbiontics is called glycosylation independent lysosomal targeting. It is meant to make enzyme-replacement therapies more effective. Replacement enzymes need to attach to target cells in the body – if they do not, they are flushed out. Symbiontics’ targeting system was designed to make the enzymes attach more tenaciously.

ZyStor’s offices, at the Milwaukee County Research Park, are also under construction and should be ready by January, the company said.