GENE THERAPY PLAYERS
An FT Pharmaceuticals Management Report
(1997)
TABLE OF CONTENTS
EXECUTIVE SUMMARY
PART 1
OVERVIEW OF COMMERCIAL ACTIVITY IN THE FIELD OF GENE THERAPY
General introduction
Established and new approaches to gene therapy
Ex vivo gene therapy
In vivo gene therapy
Target diseases for gene therapy
Monogenic diseases
Polygenic and acquired diseases
Diseases currently treated by therapeutic protein
administration
Gene therapy versus other forms of oligonucleotide therapy
Gene delivery strategies
Virus-derived gene delivery vectors
Vectors derived from retroviruses
Vectors derived from adenoviruses, adenoassociated
viruses and herpes simplex virus type 1
Novel virus-derived vectors
Nonviral gene delivery vectors
Chemical methods of delivery
Physical methods of delivery
Receptor-mediated gene delivery
Second-generation gene therapy: vector design for targeted
integration within the genome
Mammalian artificial chromosomes and other novel approaches
to gene delivery
Strategies for controlling gene expression
Cells as gene vectors
Stem cells as gene vectors
Differentiated cells as cell vectors
Protection of transplanted cells from the host's immune
system
Gene therapy approaches to treatment of monogenic diseases
Gene therapy approaches to treatment of cancer
Gene therapy as an aid to conventional chemotherapy
Viral prodrug activating therapy
Introduction of multidrug resistance genes into
noncancerous cells
Transgenic immunotherapy for cancer
Cytokine immunotherapy
Immunotherapy based on tumour-associated antigens
Modification of expression of tumour suppressor genes and
oncogenes
Replacement of defective tumour suppressor genes
Suppression of oncogene expression
Additional gene therapy approaches under investigation
Gene therapy approaches to treatment of polygenic and acquired
diseases
Cardiovascular disease
Disorders of the nervous system
Other therapeutic applications of gene therapy in commercial
development
Gene therapy approaches to treatment and prevention of
infectious diseases
Immunotherapeutic gene transfer products
Genomics companies with gene therapy programmes
Companies offering support services to gene therapy companies
Commercialisation of gene therapy
Gene therapy as a service business
Gene therapy involving disease-specific pharmaceutical
products
Business strategies
Strategies for product development and clinical
testing
Strategies for selecting gene transfer systems for
development
Strategies for disease-target selection
PART 2
COMPANY PROFILES
Company index
Information relating to recent acquisitions, mergers and
name changes
SECTION 1: MAJOR DEVELOPERS OF GENE THERAPIES AND RELATED
TECHNOLOGIES
Aastrom Biosciences Inc
Advanced Therapies Inc
Alexion Pharmaceuticals Inc
AntiCancer Inc
Apollon Inc
Ariad Pharmaceuticals Inc/Ariad Gene Therapeutics
Avigen Inc
Aviron Inc
Biohybrid Technologies Inc
Cell Genesys Inc
Chiron Corp/Chiron Viagene
Chromos Molecular Systems Inc
Codon Pharmaceuticals Inc
CytoTherapeutics Inc
Dendritech Inc
Epoch Pharmaceuticals Inc
GeneMedicine Inc
Genovo Inc
GenVec Inc
Genzyme Corp
Geron Corporation
Human Genome Sciences Inc
Immune Response Corporation, The
Immusol Inc
Inex Pharmaceuticals Corp
Ingenex Inc
IntraImmune Therapies Inc
Introgen Therapeutics Inc
Introgene BV
Kimeragen
Megabios Corp
NeuroVir Research Inc
NuGene Technologies Inc
Osiris Therapeutics Inc
Oxford BioMedica plc
Progenitor Inc
Somatix Therapy Corp
TargeTech
Targeted Genetics Corp
Therexsys Ltd
Transgene
Transkaryotic Therapies Inc
Vical Inc
SECTION 2: MULTINATIONALS WITH KNOWN INTERESTS IN GENE THERAPY
AND RELATED AREAS
American Home Products Corp
Wyeth-Ayerst Laboratories
Amgen Inc
Astra AB
Baxter International Inc/Baxter Healthcare
Bayer AG
Boehringer Ingelheim GmbH
Bristol-Myers Squibb Co
Corange International Ltd
Boehringer Mannheim GmbH
Du Pont Merck Pharmaceutical Co
Genentech Inc
Genetics Institute Inc
Glaxo Wellcome plc
Hoechst Marion Roussel
Merck & Co
Novartis Pharma AG
Genetic Therapy Inc
SyStemix
Pasteur Merieux
Pfizer Inc/Pfizergen
Pharmacia & Upjohn Inc
Procter & Gamble Co
Rhone-Poulenc Rorer SA
Applied Immune Sciences Inc
ExVivo Therapies
Roche Holdings AG
Syntex
RPR Gencell
Schering-Plough Corp
Canji Inc
SmithKline Beecham Plc
Warner-Lambert Co
W.R.Grace & CO
Auragen
SECTION 3: COMPANIES WITH AN EARLY-STAGE OR LIMITED INVOLVEMENT
IN GENE THERAPY AND RELATED AREAS
Acorda Therapeutics Inc
Activated Cell Therapy Inc
Advanced Magnetics Inc
Advanced Tissue Sciences (ATS) Inc
Akzo Fazer AG Membrana
Amcell Corp
Aronex Pharmaceuticals Inc
Athersys Inc
Bavarian Nordic Research Institute A/S
Biocompatibles International plc
Biocyte Corporation
Biogen Inc
Biovector Therapeutics
Boston Scientific Corp
British Biotechnology plc
British Technology Group plc
Calydon Inc
Cantab Pharmaceuticals plc
Cellcor Inc
CellGenix
CellPro Inc
CellStat Technologies
Celltech Group plc
Clestra Cleanroom Inc
Clonexpress
Copernicus Gene Systems Inc
Cor Therapeutics Inc
Cortecs International plc
Cryo-Cell International
Cytel Corporation
Darwin Molecular Corp
Dnavec Research
DNAX Research Institute
Dynal AS
Enzon Inc
Genebiomed Inc
Geneic Sciences Inc
Genetix Pharmaceuticals Inc
Geniva
Genopoietic SA
Gilead Sciences Inc
Green Cross Corporation, The
Gryphon Sciences
Hadasit
Imclone Systems Inc
Immunex Corporation
Imperial Cancer Research Technology Ltd
Laboratoires Fournier SA
Magenta Corp
Medarex Inc
MediGene GmbH
Medi-Ject
Neocrin Co
Neurotech
NovoNordisk A/S
Ontogeny Inc
Orthogen
Pharmadigm Inc
PharmaGenics Inc
Protologics Corp
Qiagen NV
Ravizza Farmaceutici
Research Corporation Technologies Inc
Sennes Drug Innovations
SRI Internnational
Stem Cell Sciences Inc
StressGen Biotechnologies Corporation
Sugen Inc
Takara Shuzo Co
Theracell Inc
Theratechnologies Inc
Therion Biologics Corp
Titan Pharmaceuticals Inc
Transcell Technologies inc
Vertex Pharmaceuticals Inc
Vion Pharmaceuticals Inc
Virogenetics Corp
VivoRx Inc
GLOSSARY
Technical terms and abbreviations
Terms and abbreviations used in drug development and marketing
LIST OF TABLES
Table 1 Companies known to be developing retroviral vector
technologies
Table 2 Comparison of virus-derived gene transfer vectors
Table 3 Companies known to be developing adenoviral, AAV and
HSV vector technologies
Table 4 Companies developing novel viral vectors
Table 5 Companies developing chemical methods of gene delivery
Table 6 Companies developing physical methods of gene delivery
Table 7 Companies developing receptor-based systems for
targeted delivery to cells and tissues
Table 8 Companies developing systems which allow direct gene
modification (repair, activation or inactivation)
Table 9 Companies developing novel nonviral delivery vectors
Table 10 Companies developing strategies for controlling gene
expression
Table 11 Companies active in the stem cell purification and
modification business
Table 12 Miscellaneous businesses allied to stem cell therapies
Table 13 Companies developing differentiated cells for use in
gene therapy
Table 14 Companies developing technologies to protect
transplanted cells from the host's immune system
Table 15 Gene therapies for monogenic diseases under commercial
development
Table 16 Companies developing gene therapy as an aid to
conventional chemotherapy
Table 17 Companies developing transgenic immunotherapy for
cancer
Table 18 Companies attempting replacement of defective tumour
suppressor genes or suppression of oncogene expression
Table 19 Novel approaches to gene therapy for cancer under
commercial development
Table 20 Companies developing gene therapies for treatment of
cardiovascular disease
Table 21 Companies developing cell and gene therapies for
treatment of disorders of the nervous system
Table 22 Companies developing gene therapies for miscellaneous
diseases
Table 23 Companies developing gene therapy approaches to
treatment and prevention of AIDS
Table 24 Companies developing gene therapy approaches to
prevention and in some cases treatment of infectious
diseases other than Aids
Table 25 Genomics companies with gene therapy programmes
Table 26 Companies offering support services to gene therapy
companies
Table 27 Examples of approaches to marketing of gene therapy
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