Enzyme based therapies
Enzyme based therapies therapy aims to treat patients by replacing a specific enzyme that is deficient or absent.
A large class of genetic diseases, also called inborn errors of metabolism, are due to the defects of single genes that code for enzymes which facilitate conversion of various substrates into other products. In most such disorders, problems arise due to accumulation of substances which are toxic of interfere with normal function, or to the effects of reduced ability to synthesize essential compounds.
The clinical manifestations of these diseases are extremely variable ranging from severe debilitating, lethal diseases in early infancy, to attenuated presentations in late adulthood. Though rare, they can have devastating consequences for patients and their families. Erytech is studying the use of ERYCAPS technology to promote long-acting enzyme activity which we believe may result in attractive product development opportunities in the enzyme based therapies.