High Value Fermentation Products, Volume 1

Chapter 10Enzymes as Therapeutic Agents in Human Disease Management

Babbal, Adivitiya, Shilpa Mohanty and Yogender Pal Khasa^*

Department of Microbiology, University of Delhi South Campus, New Delhi, (India)

*Corresponding author: [e-mail]

Abstract

Enzymes are biological macromolecules that can be used as biotherapeutic agents due to high specificity, affinity and efficacy. They are commonly used for the treatment of diseases like Alzheimer’s, lymhphoblastic leukemia, refractory gout, lysosomal storage disorders, cardiovascular diseases and pancreatic disorders. Moreover, the introduction of the Orphan Drug Act allowed the development of enzyme-based drugs for the treatment of rare genetic disorders which resulted in the improvement of patients’ quality of life with significant reduction in morbidity. Further, the techniques like PEGylation, release of therapeutic molecules at the target site, polymer-enzyme conjugates, nano-immobilization and masking of antigenic region have been employed to design drugs with a high potency and stability. Several enzyme based bio-therapeutics have been approved by FDA and many are in clinical trials. The advancement in recombinant DNA technology has further helped in the development of efficient disease-specific enzyme-based therapies cost-effectively.

Keywords: Therapeutic enzymes, orphan drugs, immunogenicity, enzyme replacement therapy, Food and Drug Administration (FDA)

10.1 Introduction

The use of enzymes as therapeutic molecules ...