GENE THERAPY PLAYERS
A FINANCIAL TIMES BIOFRONTIERS REPORT
(1999)
TABLE OF CONTENTS
EXECUTIVE SUMMARY
CHAPTER 1 MARKET AND TECHNOLOGY OVERVIEW
INTRODUCTION AND SCOPE
TRADITIONAL 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 DELIVERY STRATEGIES
Virus-derived gene delivery vectors
Nonviral gene delivery vectors
Chemical methods of gene delivery
Physical methods of gene delivery
Targeted gene delivery
Novel approaches to gene delivery
STRATEGIES FOR CONTROLLING GENE EXPRESSION
CELLS AS GENE VECTORS
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 of cancer
Cytokine immunotherapy
Immunotherapy based on tumour-associated antigens
Replacement of defective tumour suppressor genes
Additional gene therapy approaches under investigation
GENE THERAPY APPROACHES TO TREATMENT OF CARDIOVASCULAR DISEASE
GENE THERAPY APPROACHES TO TREATMENT OF DISORDERS
OF THE NERVOUS SYSTEM
GENE THERAPY APPROACHES TO TREATMENT AND PREVENTION
OF INFECTIOUS DISEASES
OTHER THERAPEUTIC APPLICATIONS OF GENE THERAPY
IN COMMERCIAL DEVELOPMENT
CHAPTER 2 COMPANY PROFILES
AASTROM BIOSCIENCES INC
ACCESS PHARMACEUTICALS INC
ACORDA THERAPEUTICS
ADVANCED THERAPIES INC
ALEXION PHARMACEUTICALS INC
ALFACELL CORP
ALPHAVAX
AMCELL CORP
AMERICAN HOME PRODUCTS
AMRAD CORPORATION LTD
ANGIOGENE PHARMACEUTICALS
ANTICANCER INC
ARADIGM CORPORATION
ARIAD PHARMACEUTICALS INC
ATHERSYS
AVIGEN INC
AVIRON INC
BAVARIAN NORDIC RESEARCH INSTITUTE A/S
BAYER AG
BIOCYTE CORP
BIODELIVERY SCIENCES INC
BIOGEN INC
BIORELIANCE CORP
BIOTRANSPLANT INC
BIOVECTOR THERAPEUTICS
BOEHRINGER INGELHEIM
BOSTON LIFE SCIENCES INC
BOSTON SCIENTIFIC CORP
BRIANA BIO-TECH
BRITISH TECHNOLOGY GROUP PLC
CALYDON INC
CANTAB PHARMACEUTICALS PLC
CELL GENESYS INC
CELLPRO INC
CELLTEC GmbH
CELSION CORP
CHIRON
CHROMOS MOLECULAR SYSTEMS Inc
COBRA THERAPEUTICS LTD
COLLATERAL THERAPEUTICS INC
COPERNICUS THERAPEUTICS INC
CORTECS INTERNATIONAL LTD
CYTOTHERAPEUTICS INC
DENDRITECH
DEPOTECH CORPORATION
DEPUY INC
DIAGNOCURE INC
DYNAL A.S.
ENDOCYTE INC
ENZO BIOCHEM INC
ENZON INC
EUROGENE LTD
FAULDING, F H & CO LTD
FeRx INC
GENELABS TECHNOLOGIES INC
GENETIX PHARMACEUTICALS INC
GENETIC REPAIR SYSTEMS
GENETRONICS BIOMEDICAL
GENOPOIETIC
GENOVO
GENVEC INC
GENZYME CORPORATION
GERON Corporation
GLAXO WELLCOME
GRYPHON SCIENCES
HEMOSOL INC
HOECHST AG (AVENTIS)
HUMAN GENOME SCIENCES INC
HYAL PHARMACEUTICAL CORPORATION
IMARx PHARMACEUTICAL CORPORATION
IMCLONE SYSTEMS INC
IMMUNE RESPONSE CORPORATION, THE
IMMUSOL INC
INEX PHARMACEUTICALS CORPORATION
INKINE PHARMACEUTICAL CO INC
INTERCARDIA INC
INTRACEL CORP
INTRAIMMUNE THERAPIES INC
INTROGEN THERAPEUTICS INC
INTROGENE BV
INTRONN LLC
JOHNSON & JOHNSON
LABORATOIRES FOURNIER S.C.A
LILLY ELI & COMPANY
LIPOSOME COMPANY INC, THE
LXR BIOTECHNOLOGY INC
MAINGEN
MEDEVA PLC
MEDIGENE GmbH
MEDI-JECT CORP
MERCK & Co
MIRUS CORP
MITOTIX INC
NATURE TECHNOLOGY CORP
NEUROTECH
NEUROVEX
NEUROVIR
NEXELL THERAPEUTICS INC
NOVARTIS AG
NUGENE TECHNOLOGIES INC
OCTOPLUS B.V.
ONCORMED INC
ONTOGENY INC
ORGANOGENESIS INC
OSIRIS THERAPEUTICS Inc
OXFORD BIOMEDICA PLC
PACIFIC PHARMACEUTICALS INC
PHARMADIGM INC
POLYMASC PHARMACEUTICALS PLC
POWDERJECT PHARMACEUTICALS PLC
REPLICOR INC
RESEARCH CORPORATION TECHNOLOGIES INC
RHONE-POULENC RORER SA (RPR) (AVENTIS)
RIBOZYME PHARMACEUTICALS INC
ROCHE HOLDING LTD
SCHERING AG
SCHERING-PLOUGH CORPORATION
SELECTIVE GENETICS INC
SENNES DRUG INNOVATIONS
SEQUUS PHARMACEUTICALS INC
SMITHKLINE BEECHAM PLC
STRESSGEN BIOTECHNOLOGIES CORPORATION
TARGETED GENETICS CORPORATION
THERATECHNOLOGIES
THERION BIOLOGICS CORP
TITAN PHARMACEUTICALS INC
TRANSGENE
TRANSKARYOTIC THERAPIES
TRANZYME PHARMACEUTICALS INC
TRIMEDYNE
UNITED THERAPEUTICS CORP
UROGEN CORP
VALENTIS INC
VASCULAR GENETICS INC
VICAL
VION PHARMACEUTICALS INC
VIVUS INC
WARNER-LAMBERT COMPANY
CHAPTER 3 FINANCIAL AND MARKET ANALYSES
Markets for gene therapy
Table 3.1 Important clinical indications for gene therapy
Table 3.2 World Pharmaceutical Market by Region in 1998
Table 3.3 Sizes of gene therapy-related Pharma Markets
in 1998
Table 3.4 Gene therapy products in development
Table 3.5 Gene therapy market in 1999 and 2005
Table 3.6 Top 70 Companies Ranked by Total Revenues
Table 3.7 Top 70 Companies Ranked by Net Income/Loss
Table 3.8 Top 70 Companies Ranked by Total Assets
Table 3.9 Top 70 Companies Ranked by R&D Expenditure
CHAPTER 4 TRENDS AND OPPORTUNITIES
Disappointments bring about a paradigm change
Traditional gene therapy promised permanent cures
Successes pave the way for stem cell therapies
Transient gene therapy offers a new way of
delivering therapeutic proteins
Promising new therapy targets for transient gene therapy
Cardiovascular disease
Tissue repair and regeneration
Control of expression is the next goal
First attempt is made to deliver gene therapy orally
Gene therapy will offer an adjunct to traditional treatments
First steps are taken towards systemic delivery of cancer gene
therapy
Bright future predicted for gene vaccines
Gene therapy will be needed early in HIV infection
Monogenic diseases continue to be targets for gene therapy
Cystic fibrosis gene therapy looks promising
Collaboration not competition is the key to success
Gene Therapy Patents - subject matter and assignees
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