Regeneron Pharmaceuticals, Inc.
COMPOSITIONS AND METHODS FOR INTERNALIZING ENZYMES

Abstract:

Compositions and methods for treating enzyme-deficiency diseases are disclosed. Multidomain therapeutic proteins containing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The multidomain therapeutic proteins are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.