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.