Muscling in on Myozyme

Oxyrane U.K. Ltd. is developing a next-generation enzyme replacement therapy for Pompe's disease that could improve on Myozyme. By enhancing delivery of enzyme to diseased tissue, the company hopes to reduce dosage and infusion times and lower cost to patients and payers.

Pompe's is an inherited disorder of glycogen metabolism caused by an absence of or deficiency in the lysosomal enzyme acid alpha glucosidase (GAA). The resulting accumulation of glycogen in tissues, especially cardiac and skeletal muscle, leads to severe and progressive muscle weakness, cardiomyopathy and respiratory failure. The disease has an estimated incidence of 1 in 40,000...