Therapeutic enzymes exhibit fascinating features and opportunities, and represent a significant and promising subcategory of modern biopharmaceuticals for the treatment of several severe diseases. Research and drug developments efforts and the advancements in biotechnology over the past twenty years have greatly assisted the introduction of efficient and safe enzyme-based therapies for a range of both rare and common disorders. The introduction and regulatory approval of twenty different recombinant enzymes has enabled effective enzyme-replacement therapy. This volume aims to overview these therapeutic enzymes, focusing in particular on more recently approved enzymes produced by recombinant DNA technology. This volume is composed of four sections. Section 1 provides an overview of the production process and biochemical characterization of therapeutic enzymes, while Section 2 focuses upon the engineering strategies and delivery methods of therapeutic enzymes. Section 3 highlights the clinical applications of approved therapeutic enzymes, including aspects on their structure, indications and mechanisms of action. Together with information on these mechanisms, safety and immunogenicity issues and various adverse events of the recombinant enzymes used for therapy are discussed. Section 4, provides discussion on the prospective and future developments of new therapeutic enzymes. This book is aimed at academics, researchers and students undertaking advanced undergraduate/postgraduate programs in the biopharmaceutical/biotechnology area who wish to gain a comprehensive understanding of enzyme-based therapeutic molecules.

Incorporates a wealth of research that clarifies several aspects of the development and application of therapeutic enzymes Equips readers with a comprehensive understanding of enzyme-based therapeutic molecules Emphasizes three areas of recombinant therapeutic enzymes and their clinical and pharmaceutical technology

Klappentext
This edited work presents studies that clarify several aspects of the development and application of therapeutic enzymes. Therapeutic enzymes exhibit fascinating features and opportunities, and represent a significant and promising subcategory of modern biopharmaceuticals for the treatment of several severe diseases. Research and drug developments efforts and the advancements in biotechnology over the past twenty years have greatly assisted the introduction of efficient and safe enzyme-based therapies for a range of both rare and common disorders. The introduction and regulatory approval of twenty different recombinant enzymes has enabled effective enzyme-replacement therapy.

This book covers mainly three areas of recombinant therapeutic enzymes and their clinical and pharmaceutical technology: (i) overview of the production process and biochemical characterization of therapeutic enzymes, (ii) focuses upon the engineering strategies and delivery methods of therapeutic enzymes,(iii) clinical applications of selected therapeutic enzymes, including aspects on their mechanisms of action and information on safety, immunogenicity issues and various adverse events of the enzymes used for therapy.

The topic of this book is particularly relevant to academics, researchers and students undertaking advanced undergraduate/postgraduate programs in the biopharmaceutical/biotechnology area who wish to gain a comprehensive understanding of enzyme-based therapeutic molecules.

Inhalt

Chapter 1. Introduction to therapeutic enzymes.- Chapter 2. Upstream processing technologies.- Chapter 3. Downstream processing technologies.- Chapter 4. Enzyme Manufacturing Process and Quality Control Evaluations.- Chapter 5. Regulatory guidance in the production of therapeutic enzymes.- Chapter 6. Biophysical methods for the characterization of Enzyme Therapeutics Bertrand Raynal Patrick England.- Chapter 7. Clinical Development of a therapeutic enzyme.- Chapter 8. Chemical modifications of therapeutic proteins.- Chapter 9. Formulation of therapeutic enzymes.- Chapter 10. Engineering of therapeutic enzymes for improving activity.- Chapter 11. Engineering of therapeutic enzymes for improving stability.- Chapter 12. Design and engineering of deimmunized therapeutic enzymes.- Chapter 13. Nanoparticles for delivery of therapeutic enzymes.- Chapter 14. Targeted delivery of therapeutic enzymes.- Chapter 15. Oral and inhalable enzyme therapies.- Chapter 16. Regulatory issues.- Chapter 17. Enzyme replacement therapies.- Chapter 18. Pharmacodynamics and pharmacokinetics issues of enzyme replacement therapy.- Chapter 19. Enzyme therapy for lysosomal storage disorders.- Chapter 20. Enzyme therapy for myocardial infarction.- Chapter 21. Enzyme therapy for chronic gout.- Chapter 22. Enzyme therapy for tumor lysis syndrome.- Chapter 23. Enzyme therapy for collagen-based disorders.- Chapter 24. Enzyme therapy for severe combined immunodeficiency disease.- Chapter 25. Enzyme therapy for detoxification of methotrexate.- Chapter 26. Enzyme therapy vitreomacular adhesion.- Chapter 27. L-Asparaginase as anticancer agent.- Chapter 28. Pancreatic enzyme replacement therapy.- Chapter 29. Enzymes in metabolic anticancer therapy.- Chapter 30. L-Arginase for L-Arg depletion therapy incancer.- Chapter 31. Methionine L-lyase for L-Met depletion therapy in cancer.- Chapter 32. Enzyme therapy for celiac sprue.- Chapter 33. Acid ceramidase.- Chapter 34. Lysosomal acid lipase.- Chapter 35. Alkaline phosphatase and hypophosphatasia.- Chapter 36. Enzybiotics: Antibiotic enzymes as drugs and therapeutics.- Chapter 37. Anti-inflammatory enzymes.- Chapter 38. Phenylalanine ammonia lyase for the treatment of phenylketonuria.- Chapter 39. Clinical applications of Hyaluronidase.- Chapter 40. Superoxide dismutase and oxidative stress modulation.- Chapter 41. Organophosphate hydrolases as catalytic bioscavengers.- Chapter 42. Novel therapeutic proteases.- Chapter 43. Genome-editing enzymes.- Chapter 44. Gene therapy for enzyme deficiency: current status and future prospects.