Enzyme based therapies aim 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 or interfere with normal function, or are the effects of the body’s reduced ability to synthesize essential compounds.
Though rare, they can have devastating consequences for patients and their families. We are studying the use of ERYCAPS® technology to promote long-acting enzyme activity, which we believe may result in attractive product development or partnering opportunities for enzyme-based therapies.