Biotechnology: A: Recombinant Proteins, Monoclonal Antibodies, and Therapeutic Genes, Volume 5a, Second Edition

564 Pages·1999·24.32 MB·English

Structure and Function of Proteins
Biotechnology
Second Edition
Volume 5a
Recombinant Proteins, Monoclonal
Antibodies and Therapeutic Genes
8
WILEYWCH
Biotechnology
Second Edition
Fundamentals Special Topics
Volume 1 Volume 9
Biological Fundamentals Enzymes, Biomass, Food and Feed
Volume 2 Volume 10
Genetic Fundamentals and Special Processes
Genetic Engineering
Volumes lla and b
Volume 3 Environmental Processes
Bioprocessing
Volume 12
Volume 4 Legal, Economic and
Measuring, Modelling and Control Ethical Dimensions
Products
Volume 5a
Recombinant Proteins, Monoclonal
Antibodies and Therapeutic Genes
Volume 5b
Genomics
Volume 6
Products of Primary Metabolism
Volume 7
Products of Secondary Metabolism
Volumes 8a and b
Biotransformations I and I1
A Multi-Volume Comprehensive Treatise
Biotechnology
Second, Completely Revised Edition
Edited by
H.-J. Rehm and G. Reed
in cooperation with
A. Puhler and F? Stadler
Volume 5a
Recombinant Proteins,
Monoclonal Antibodies
and Therapeutic Genes
Edited by
A. Mountain, U. Ney and D. Schomburg
8W ILEY-VCH
Weinheim - New York . Chichester . Brisbane . Singapore Toronto
Series Editors: Volume Editors:
Prof. Dr. H.-J. Rehm Dr. G. Reed Dr. A. Mountain
Institut fur Mikrobiologie 1029 N. Jackson St. #501-A Cobra Therapeutics Ltd.
Universitat Miinster Milwaukee,W I 53202-3226 The Science Park
Corrensstralje 3 USA University of Keele
D-48149 Munster Keele, Staffordshire ST5 5SP
FRG UK
Prof. Dr. A. Puhler Pro€ Dr. P. J. W. Stadler Dr. U. Ney
Biologie VI (Genetik) Artemis Pharmaceuticals Celltech Therapeutics Ltd.
Universitat Bielefeld Geschaftsfiihrung 216 Bath Road
P.O. Box 10013 1 Pharmazentrum Koln Slough Berkshire SL14EN
D-33501 Bielefeld Neurather Ring UK
FRG D-51063 Koln
FRG Prof. Dr. D. Schomburg
Institut fur Biochemie
Universitat zu Koln
Zulpicher Str. 47
D-50674 Koln
FRG
This book was carefully produced. Nevertheless, authors, editors and publisher do not warrant the information
contained therein to be free of errors. Readers are advised to keep in mind that statements, data, illustrations,
procedural details or other items may inadvertently be inaccurate.
Library of Congress Card No.: applied for
British Library Cataloguing-in-PublicationD ata:
A catalogue record for this book is available from the British Library
Die Deutsche Bibliothek - CIP-Einheitsaufnahme
Biotechnology : a multi volume comprehensive
treatise / ed. by H.-J. Rehm and G. Reed.
In
cooperation with A. F’iihler and P. Stadler. -
2., completely rev. ed. - VCH.
ISBN 3-527-28310-2 (Weinheim ...)
NE: Rehm, Hans-J. [Hrsg.]
Vol. 5a: Recombinant Proteins, Monoclonal Antibodies and Therapeutic Genes / ed. by A. Mountain, U. Ney
and D. Schomburg - 1999
ISBN 3-527-28315-3
0 WILEY-VCH Verlag GmbH, D-69469 Weinheim (Federal Republic of Germany), 1999
Printed on acid-free and chlorine-free paper.
All rights reserved (including those of translation into other languages). No part of this book may be reproduced in any
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Bookbinding:J . Schaffer, D-67269 Griinstadt.
Printed in the Federal Republic of Germany
Preface
In recognition of the enormous advances in series. Its members come from key institu-
biotechnology in recent years, we are pleased tions representing scientific input from about
to present this Second Edition of “Biotech- twenty countries.
nology” relatively soon after the introduction The volume editors and the authors of the
of the First Edition of this multi-volume com- individual chapters have been chosen for
prehensive treatise. Since this series was ex- their recognized expertise and their contribu-
tremely well accepted by the scientific com- tions to the various fields of biotechnology.
munity, we have maintained the overall goal Their willingness to impart this knowledge to
of creating a number of volumes, each de- their colleagues forms the basis of “Biotech-
voted to a certain topic, which provide scien- nology” and is gratefully acknowledged.
tists in academia, industry, and public institu- Moreover, this work could not have been
tions with a well-balanced and comprehensive brought to fruition without the foresight and
overview of this growing field. We have fully the constant and diligent support of the pub-
revised the Second Edition and expanded it lisher. We are grateful to VCH for publishing
from ten to twelve volumes in order to take “Biotechnology” with their customary excel-
all recent developments into account. lence. Special thanks are due to Dr. Hans-
These twelve volumes are organized into Joachim Kraus and Karin Dembowsky, with-
three sections. The first four volumes consid- out whose constant efforts the series could
er the fundamentals of biotechnology from not be published. Finally, the editors wish to
biological, biochemical, molecular biological, thank the members of the Scientific Advisory
and chemical engineering perspectives. The Board for their encouragement, their helpful
next four volumes are devoted to products of suggestions, and their constructive criticism.
industrial relevance. Special attention is given
here to products derived from genetically en- H.-J. Rehm
gineered microorganisms and mammalian G. Reed
cells. The last four volumes are dedicated to A. Piihler
the description of special topics. P. Stadler
The new “Biotechnology” is a reference
work, a comprehensive description of the
state-of-the-art, and a guide to the original
literature. It is specifically directed to micro-
biologists, biochemists, molecular biologists,
bioengineers, chemical engineers, and food
and pharmaceutical chemists working in indus-
try, at universities or at public institutions.
A carefully selected and distinguished
Scientific Advisory Board stands behind the
Scientific Advisory Board
Prof Dr. M. J. Beker Prof Dr. T. K. Ghose
August Kirchenstein Institute of Microbiology Biochemical Engineering Research Centre
Latvian Academy of Sciences Indian Institute of Technology
Riga, Latvia New Delhi, India
Prof Dr. J. D. Bu’Lock Prof Dr. I. Goldberg
Weizmann Microbial Chemistry Laboratory Department of Applied Microbiology
Department of Chemistry The Hebrew University
University of Manchester Jerusalem, Israel
Manchester, UK
Prof Dr. C. L. Cooney Prof Dr. G. Goma
Department of Chemical Engineering DCpartement de GCnie Biochimique et
Massachusetts Institute of Technology Alimentaire
Cambridge, MA, USA Institut National des Sciences AppliquCes
Toulouse, France
Prof Dr. H. W. Doelle Sir D. A. Hopwood
Department of Microbiology Department of Genetics
University of Queensland John Innes Institute
St. Lucia, Australia Norwich, UK
Prof Dr. J. Drews Prof Dr. E. H. Houwink
F. Hoffmann-La Roche AG Organon International bv
Basel, Switzerland Scientific Development Group
Oss, The Netherlands
Prof Dr. A. Fiechter Prof Dr. A. E. Humphrey
Institut fur Biotechnologie Center for Molecular Bioscience and
Eidgenossische Technische Hochschule Biotechnology
Zurich, Switzerland Lehigh University
Bethlehem, PA, USA
VIII Scientific Advisory Board
Prof Dr. I. Karube Prof Dr. K. Schiigerl
Research Center for Advanced Science Institut fur Technische Chemie
and Technology Universitat Hannover
University of Tokyo Hannover, Germany
Tokyo, Japan
Prof Dr. M. A. Lachance Prof Dr. P. Semi
Department of Plant Sciences Chair of Fermentation Chemistry
University of Western Ontario and Industrial Microbiology
London, Ontario, Canada Lepetit Research Center
Gerenzano, Italy
Prof Dr. Y.L iu Prof Dr. Y.H . Tan
China National Center for Biotechnology Institute of Molecular and Cell Biology
Development National University of Singapore
Beijing, China Singapore
Prof Dr. J. F. Martin Prof Dr. D. Thomas
Department of Microbiology Laboratoire de Technologie Enzymatique
University of Le6n UniversitC de Compi2gne
Leon, Spain Compikgne, France
Prof Dr. B. Mattiasson Prof Dr. W. Verstraete
Department of Biotechnology Laboratory of Microbial Ecology
Chemical Center Rijksuniversiteit Gent
University of Lund Gent, Belgium
Lund, Sweden
Prof Dr. M. Roehr Prof Dr. E.-L. Winnacker
Institut fur Biochemische Technologie Institut fur Biochemie
und Mikrobiologie Universitat Munchen
Technische Universitat Wien Munchen, Germany
Wien, Austria
Prof Dr. H. Sahm
Institut fur Biotechnologie
Forschungszentrum Julich
Julich, Germany
Contents
Introduction xv Monoclonal Antibodies
U. Ney, D. Schomhurg
7 Antibody Engineering
and Expression 219
J. R. Adair
Structure and Function of Proteins 8 Manufacture of Therapeutic
Antibodies 245
A.J . Racher, J. M. Tong, J. Bonnerjea
1 Types and Function of Proteins 1
9 Use of Antibodies for Immuno-
S. Wolt;H . G. Gassen
purification 275
2 Sequence and Structure of Proteins 43
D. J. King
i? Eisenhaber, P. Bork
10 Preclinical Testing of Antibodies:
3 Protein Interactions 87
Pharmacology, Kinetics,
M. Meyer, D. Schonzhurg
and Immunogenicity 289
R. Foulkes, Stephens
S.
11 Preclinical Testing of Antibodies:
Safety Aspects 303
Recombinant Proteins R. W James
12 Therapeutic Applications
4 In vitro Folding of Monoclonal Antibodies:
of Inclusion Body Proteins A Clinical Overview 311
on an Industrial Scale 111 M. Sopwith
R. Rudolph, H. Lilie, E. Schwarz
5 Medical Applications
of Recombinant Proteins
in Humans and Animals 125 Case Studies
G. D. Wetzel
6 Enzymes for Industrial Applications 189
13 Antibodies for Sepsis:
W Aehle. 0. Misset
Some Lessons Learnt 329
M. Opal
S.
14 An Engineered Human Antibody
for Chronic Therapy: CDP571 343
M. Sopwith, S. Stephens
15 Antibody Targeted Chemotherapy 355
M. S. Berger, P. R. Hamann, M. Sopwith
X Contents
16 ReoPro Clinical Development: Regulatory and Economic Aspects
A Case Study 365
H. E Weisman 22 Regulation of Antibodies
and Recombinant Proteins 495
J. Foulkes, G. Traynor
23 Regulation of Human Gene Therapy 517
Gene Therapy J. Parker
24 Economic Considerations 531
17 Overview of Gene Therapy 383 I. J. Nicholson
A. Mountain
18 Viral Vectors for Gene Therapy 395
B. J. Carter
19 Non-Viral Vectors for Gene Therapy 427 Index 543
N Weir
20 Issues for Large-Scale Plasmid DNA
Manufacturing 443
M. Schleef
21 Gene Therapy for HIV Infection 471
M. Poznansky, Myra McClure,
Gregor B. Adams