Monday, March 5, 2001
The point of doing targeted therapy is that it generally requires a smaller amount of medication compared to systemic therapy, which relies on diffusion of drug to the site of action. Novazyme Inc. is trying to address the lysosomal storage diseases by using its glycobiology technology to target needed enzymes to their sites of action. By targeting the products, Novazyme hopes to reduce the amount of enzyme required and reduce side effects.
There are 49 lysosomal storage diseases, including Pompe's disease, the Mucopolysaccharidoses (MPS) I-IV, Gaucher disease, and Fabry disease. All of the diseases are inherited as either autosomal recessive traits or as X-chromosome-linked traits.