Gene Therapy

Published by: Global Industry Analysts

Published: Feb. 1, 2006 - 415 Pages

Table of Contents

I. INTRODUCTION, METHODOLOGY & PRODUCT DEFINITIONS

II. EXECUTIVE SUMMARY

1. Market Dynamics

Future-On a Promising Note

Market Predictions Thrive On

China-First to Hit the Market

Counting on the Success Factors

Global Market by Region

US-Largest Market

Rest of World-Growing Rapidly

2. Key Issues/ Trends

Factors Influencing Gene Therapy Future Development

Human Genome Analysis

Diagnostic Industry Moves

Public Support for Gene Therapy

Factors Hampering Market Growth

Identifying Genetic Fault

Immune Response

Delivering Genetic Materials into Patient Cells

Multigene Disorders

Side Effects

Push Towards Germline Gene Transfer

3. The Global Landscape

Alliances and Acquisitions-Strategy to Success

Companies and Partnerships

Patent Pooling: Yet Another Obstacle

Commercialization: A Road Uphill

Time Line in Gene Therapy Drug Development

Target Diseases and Players Involved

Key Players by Application Area

Table 1: Gene Therapy Clinical Trials Worldwide (2004): Percentage Breakdown by Disease/ Application Area for Cancer, Cystic Fibrosis, Heart Disease, HIV, and Others (Includes Corresponding Graph/Chart)

Table 2: Gene Therapy Clinical Trials Worldwide (2004): Percentage Breakdown by Gene Type Transferred for Cytokine, Antigen, Tumor Suppressor, Suicide, Deficiency, Drug Resistance, and Others (Includes Corresponding Graph/Chart)

Table 3: Gene Therapy Clinical Trials Worldwide (2004): A Split by Development Phase (Includes Corresponding Graph/Chart)

Table 4: Gene Therapy Clinical Trials (2004): Percentage Breakdown by Geographic Region for US, Canada, Japan, Europe and Rest of World (Includes Corresponding Graph/Chart)

Various Gene Therapy Products Under Development (2005)

Gene Therapy Industry in Europe and the US-A Comparison

4. Gene Therapy-An Insight

Molecular Basics-The Gene

Genome-The Information Pool

Functional cloning

Random sequencing

Positional cloning.

What is Gene Therapy?

A Backdrop

1990 Highlights-The Preliminary Gene Therapy Trial

1995 Highlights

2000 Highlights-Clinical Trials Reaches 400-Milestone

2004 Witnesses the First Gene Therapy Drug

Classical Theory of Gene Therapy

Ex-Vivo Approach of Gene Therapy

In-vivo Approach of Gene Therapy

Types of Gene Therapy

Monogenic Gene Therapy

Suicide Gene Therapy

Antisense Gene Therapy

Various Approaches

5. Components of Gene Therapy

Vector

Delivery Vehicles and Challenges in Use of Viral Vectors

Gene Delivery-Without Virus

Recent Release of Non-Viral Products

Well-known Delivery Approaches for Gene Therapy

Viral and Non-viral Vectors in Use

Retroviruses

Adenoviruses

Adenoassociated viruses

Herpes simplex viruses

Lentiviruses

Liposomes

Viral Vectors by Companies involved

Gene Cassette

Target cells

Stem cells

Differentiated cells

The Disc: An Excellent Target for Gene Therapy

6. Review of Various Approaches

Antisense Technology

Catalytic Ribozyme Technology

Triple Helix DNA

7. Gene Therapy For Disease Treatment

Gene Therapy-A Way to Fight or Cure Genetic Disease?

Gene Therapy for Cancer Treatment

Gene Transfer and Gene Therapeutic Concepts in Cancer

Competitive Landscape in Cancer Gene Therapy

Developments in Cancer Gene Therapy

P53 Gene Therapy-A Novel Approach in Cancer Treatment

p53 Cancer Gene Therapy Drugs in Development (2004)

Gendicine-From Concept to Reality

Vaccines

Cancer Treatment with Gene Therapy and Radiation Therapy

Gene Therapy for Cardiovascular Disease

Cardiovascular Gene Therapy Products under Trial

Gene Therapy for Cystic Fibrosis Disease

Gene Therapy for Haemophilia B

Companies Engaged in Developing Gene Therapy Drugs against Haemophilia

Gene Therapy for HIV/AIDS

Gene Therapy for Parkinson’s Disease

The Disease

Therapy

8. Regulatory Environment

Gene therapy and Regulatory Environment

Gene Patents-An Overview

Regulations Governing Gene Therapy by Region

The US

Food and Drug Administration (FDA)

FDA Requires Additional Toxicology and Biodistribution Studies

CBER

NIH

RAC

Role of NIH

IRB and IBC

Outline description of approval process of FDA and NIH

European Union

France

United Kingdom

Local Research Ethics Committee (LREC)

Medicines Control Agency

Germany

Switzerland

Austria

Italy

Sweden

The Netherlands

Belgium

Spain

Japan

9. Research and Development

AMT Develops (AAV) Vector-Based Gene Therapy

Ark Comes Up with New Gene Thearpy Delivery Technology

Oxford University to Come Up with Gene Therapy for Hypertension

Asklepios to Develop New Gene Therapy

AGTC to Develop Gene Therapy for Heart Failure

10. Recent Industry Activity

Diamyd Acquires Nurel

Oxford BioMedica Accords with Sigma-Alrdrich

Oxford BioMedica Inks Licensing Agreement with Pfizer

genOway Signs Two General Service Agreements

genOway Acquires IngenKO Pty Ltd

genOway Germany Participates in EU 6th Framework Program

Crucell Strikes a Deal with Two Asian Firms

Corautus’ VEGF-2 Gene Therapy Gains Recognition from US FDA

ViroMed Ceases Development of Gene Therapy

Corautus Genetics Pacts with CSEMC

Takara Bio Collaborates with Mie University

Celladon Accords with V-Kardia

Genzyme Acquires Ilex Oncology

Genzyme and AGTC Form Collaboration

Sanofi-Synthelabo Merges with Aventis

Guidant Forms Collaboration with Columbia and Stony Brook

Avigen Concludes Collaboration with Bayer

Louisiana and Bionomics Team-Up

Avigen Receives FDA Clearance to Commence Phase I/II Clinical Trial of Av201

Oxford BioMedica and Viragen Enters into Licensing Agreement

Oxford BioMedica Signs Licensing Agreement with Merck

Avigen Starts Gene Therapy Trial for Parkinson’s Disease

Oxford BioMedica Acquires Chiron Patents

UA duo Establishes Gene-therapy Firm

SiBiono Commences Construction of Gene Therapy Facility

Avigen Receives FDA Approval for Clinical Trial of AV201

UK GTAC Approves Clinical Trial

CYGEE Bags patent Approval for Novel Gene Therapy Technology

Mirus Bio Receives $1.26 million SBIR Grant

CEGE Trials Novel Gene Therapy

Avigen Suspends Haemophilia Gene Therapy Trials

Oxford BioMedica and RPR Enters into Licensing Agreement

Schering AG Acquires Collateral Therapeutics

MolMed SpA Buys Rights to RectroNectin

Avigen Receives US FDA Approval

Transkaryotic Patent Case on Gene Therapy Settled

Targeted Genetics Receives Patents

Enzo Biochem Conducts Phase I Studies Related to HIV Treatment

Eurogene Acquires Oy Quattrogene

Cardion AG Acquires Tolerance Pharmaceuticals

Aventis Restructures Gene-Therapy Alliance with Introgen

Boehringer Ingelheim Austria GmbH Inks Agreement with BIA Separations

AVAX’s Genopoietic Receives Contract from French Army

Cell Genesys Establishes New Subsidiary

Aventis Pharma Creates New Gencell Unit

Chiron Divests Gene Therapy Manufacturing Facility

ViroMed Conducts Clinical Trials on Gene Therapy Drug

EG009 Gene Therapy Doubles Survival Rate of Malignant Glioma Patients in Phase I/II Clinical Trials

Genteric Receives Patent for Insulin Gene Pill System

Bayer Corp. Receives Marketing and Distribution Rights to Coagulin-B

Avigen Receives Patent for Parkinson’s Disease Gene Therapy

Genteric Receives US Patent for Gene Pill Method

Kimeragen Merges with ValiGene SA

Aventis Acquires License form Electrofect

DeveloGen Acquires Hepavec

Targeted Genetics Acquires Genovo Inc

GenStar Acquires Allegro Inc

Argonex and Rohm & Haas Company Enter into a Joint Venture

Nexell Therapeutics and Takara Shuzo Join Forces

BTG International Inks Licensing Agreement with Avigen Inc

Oxford BioMedica Awarded US Patens Concerning Retroviral And Lentiviral Systems

Genteric Receives Gene Therapy Patent

GenVec Granted Broad Gene Therapy Patent

11. Focus on Key Players

AnGes MG Inc (Japan)

Athersys Inc (US)

Avigen (US)

Canji Inc (US)

Cell Genesys

Centelion SA (France)

GenVec (US)

Genzyme Corporation (US)

Introgen Therapeutics Inc (US)

Oxford BioMedica (UK)

Shenzhen SiBiono GeneTech Co., Ltd (China)

Targeted Genetics Corp (US)

Transgene (France)

Valentis Inc. (US)

Vical (US)

12. Global Market Perspective

Table 5: World Current & Future Market Analysis for Gene Therapy by Geographic Region-US, and Rest of World Independently Analyzed with Annual Sales Figures in US$ Million for Years 2005 through 2010 (includes corresponding Graph/Chart)

Table 6: World 5-Year Perspective for Gene Therapy by Geographic Region-Percentage Breakdown of Dollar Sales for US, and Rest of World Markets for Years 2005 and 2010 (includes corresponding Graph/Chart)

III. MARKET

1. The United States

A. Market Analysis

Regulatory Environment

Food and Drug Administration

IRB and IBC

OBA

NIH

Focus on Key Players

Athersys Inc

Avigen

Canji Inc

Cell Genesys

GenVec

Genzyme Corporation

Introgen Therapeutics Inc

Targeted Genetics Corp

Valentis Inc.

Vical

Strategic Developments

B. Market Analytics

Table 7: US Current & Future Market Analysis for Gene Therapy-Annual Sales Figures in US$ Million for Years 2006 through 2010 (includes corresponding Graph/Chart)

2. Rest of World

A. Market Analysis

Review of Major Markets

Europe

Germany and UK-The Hub of European Gene Therapy Market

Table 8: Gene Therapy Clinical Trials in Europe (2004): Percentage Breakdown of Number of Trials by Country for UK, Germany, France, Belgium, Italy, Netherlands, and Others (includes corresponding Graph/Chart)

Regulatory Environment

Gene Therapy Advisory Committee

The European Society of Gene Therapy (ESGT)

Study of Regulatory Environment in Select European Countries

United Kingdom

Local Research Ethics Committee (LREC)

Medicines Control Agency

France

Germany

Italy

Austria

Sweden

The Netherlands

Belgium

Spain

Switzerland

Japan

Asia-Pacific

World's First Gene Therapy Medicine Approved in China

Prospects for Gendicine

Competition

Leading Gene Therapy Formulations under

Clinical Trials by Select Co., in China

Rest of World

Focus on Key Players

AnGes MG Inc (Japan)

Centelion SA (France)

Oxford BioMedica (UK)

Shenzhen SiBiono GeneTech Co., Ltd (China)

Transgene (France)

Strategic Developments

B. Market Analytics

Table 9: Rest of World Market for Gene Therapy-Current & Future Analysis by Annual Sales Figures in US$ Million for Years 2005 through 2010(includes corresponding Graph/Chart)

IV. COMPETITION

1. Affymetrix, Inc. (USA)

Table 10: Half-Yearly Sales Analysis: 2004-2005 (H1) (In US$ million)

Table 11: Annual Sales Analysis: 2003-2004 (In US$ million)

2. Aldevron LLC (USA)

3. AlphaVax, Inc. (USA)

4. Amaxa GmbH (Germany)

5. Amgen, Inc. (USA)

Table 12: Half Yearly Sales Analysis: 2004-2005 (H1) (In US$ million)

Table 13: Half Yearly Sales Analysis by Geographic Region: 2004-2005 (Q1) (In US$ million)

Table 14: Annual Sales Analysis: 2003-2004 (In US$ million)

Table 15: Annual Sales Analysis by Geographic Region: 2003-2004 (In US$ million)

6. AMT BV (The Netherlands)

7. AnGes MG, Inc. (Japan)

8. Antigenics, Inc. (USA)

Table 16: Half-Yearly Sales Analysis: 2004-2005 (H1) (In US$ million)

Table 17: Annual Sales Analysis: 2003-2004 (In US$ million)

9. Antisense Pharma GmbH (Germany)

10. Applied Genetic Technologies Corporation (USA)

11. Ariad Gene Therapeutics, Inc. (USA)

12. Ark Therapeutics Ltd. (UK)

Ark Therapeutics OY (Finland)

13. Athersys, Inc. (USA)

14. Austrianova (Austria)

15. Automated Cell Technologies, Inc. (USA)

16. Avax Technologies, Inc. (USA)

Table 18: Half Yearly Sales Analysis: 2004-2005 (H1) (In US$ thousand)

Table 19: Annual Sales Analysis: 2004-2005 (In US$ thousand)

17. AVI Biopharma, Inc. (USA)

18. Avigen, Inc. (USA)

Table 20: Half-Yearly Sales Analysis: 2004-2005 (H1) (In US$ million)

Table 21: Annual Sales Analysis: 2003-2004 (In US$ million)

Table 22: Quarterly Sales Analysis: 2003-2004 (In US $ million)

19. Bavarian Nordic A/S (Denmark)

20. Bionomics Limited (Australia)

21. Biophan Technologies, Inc. (USA)

22. Biophoenix (UK)

23. BioProtein Technologies (France)

24. Biosense Webster, Inc. (USA)

25. BioVex Ltd. (UK)

26. Celera Genomics Group (USA)

27. Cell Genesys, Inc. (USA)

Table 23: Annual Sales Analysis: 2003-2004 (In US$ million)

28. Celltech Group PLC (UK)

29. Celsion Corporation (USA)

30. Centelion (France)

31. CereGene, Inc. (USA)

32. Cobra Biomanufacturing PLC (UK)

33. Copernicus Therapeutics, Inc. (USA)

34. Corixa Corporation (USA)

Table 24: First Quarter Sales Analysis: 2004-2005 (Q1) (In US$ million)

Table 25: Annual Sales Analysis: 2003-2004 (In US$ million)

35. Cosmo SpA (Italy)

Cosmo Bioscience, Inc. (USA)

36. Crucell NV (The Netherlands)

37. Cyclacel Limited (UK)

38. Cygene Laboratories (USA)

39. Cytogen Corporation (USA)

Table 26: Half Yearly Sales Analysis: 2004-2005 (H1) (In US$ million)

Table 27: Half Yearly Sales Analysis By Product: 2004-2005 (H1) (In US$ million)

Table 28: Annual Sales Analysis: 2003-2004 (In US$ million)

Table 29: Annual Sales Analysis By Product: 2003-2004 (In US$ million)

40. CytRx Corporation (USA)

41. DeveloGen AG (Germany)

42. Diversa Corporation (USA)

43. Enzo Biochem, Inc. (USA)

44. Fit Biotech Oyj PLC (Finland)

45. Gene Therapy Systems, Inc. (USA)

46. GeneCure LLC (USA)

47. GenEra SpA (Italy)

48. Genetix Pharmaceuticals, Inc. (USA)

49. Genteric, Inc. (USA)

50. GenVec, Inc. (USA)

Table 30: Half Yearly Sales Analysis: 2004-2005 (In US$ million)

51. Genzyme Corporation (USA)

Table 31: Half Yearly Sales Analysis: 2004-2005 (H1) (In US$ million)

Table 32: Annual Sales Analysis: 2003-2004 (In US$ million)

Table 33: Annual Sales Analysis by Segment: 2003-2004 (In US$ million)

Table 34: Annual Sales Analysis by Geographical Region: 2003-2004 (In US$ million)

Genzyme Molecular Oncology (USA)

Genzyme Therapeutics (UK)

52. Geron Corporation (USA)

Table 35: First Half Sales Analysis: 2004-2005 (H1) (In US$ million)

Table 36: Annual Sales Analysis: 2003-2004 (In US$ million)

Table 37: Quarterly Sales Analysis: 2003-2004 (In US$ million)

53. Gilead Sciences, Inc. (USA)

54. Glygen Corporation (USA)

55. GTx, Inc. (USA)

56. Guidant Corporation (USA)

Table 38: Half-Yearly Sales Analysis by Quarters: 2004-2005 (H1) (In US$ million)

Table 39: Half-Yearly Sales Analysis by Product: 2004-2005 (H1) (In US$ million)

Table 40: Half-Yearly Sales Analysis by Geographic Region: 2004-2005 (H1) (In US$ million)

Table 41: Annual Sales Analysis: 2003-2004 (In US$ million)

Table 42: Annual Sales Analysis By Product: 2003-2004 (In US$ Million)

Table 43: Annual Sales Analysis By Geographic Region: 2003-2004 (In US$ million)

57. Human Genome Sciences, Inc. (USA)

Table 44: Half Yearly Sales Analysis: 2004-2005 (In US$ million)

Table 45: Annual Sales Analysis: 2003-2004 (In US$ million)

Table 46: Annual Sales Analysis by Quarters: 2003-2004 (In US$ million)

58. ImGenex Corporation (USA)

59. Immusol, Inc. (USA)

60. Innovata Plc (U K)

61. Introgen Therapeutics, Inc. (USA)

62. Intronn LLC (USA)

63. Invitrogen Corporation (USA)

Table 47: Half Yearly Sales Analysis: 2004-2005 (H1) (In US$ million)

Table 48: Annual Sales Analysis: 2003-2004 (In US$ million)

Table 49: Annual Sales Analysis by Segment: 2003-2004 (In US$ million)

64. InvivoGen (USA)

65. MaxCyte (USA)

66. Merck KGAA (Germany)

Table 50: Half Yearly Sales Analysis by Quarter: 2004-2005 (In € million)

Table 51: Annual Sales Analysis: 2003-2004 (In € million)

67. Mitsubishi Chemical Corporation (Japan)

Table 52: Annual Sales Analysis: 2003-2004 (In ¥ billion)

Table 53: Annual Sales Analysis By Segment: 2003-2004 (In ¥ billion)

68. MolMed SpA (Italy)

69. Northwest Biotherapeutics, Inc. (USA)

70. Novartis AG (Switzerland)

Table 54: Half Yearly Sales Analysis: 2004-2005 (H1) (In US$ billion)

Table 55: Annual Sales Analysis: 2003-2004 (In US$ billion)

Table 56: Annual Sales Analysis by Division: 2003-2004 (In US$ billion)

Table 57: Annual Sales Analysis by Geographical Region: 2003-2004 (In US$ billion)

Table 58: Quarterly Sales Analysis: 2003-2004 (In US$ billion)

71. NsGENE A/S (Denmark)

72. Onyx Pharmaceuticals, Inc. (USA)

73. OSI Pharmaceuticals, Inc. (USA)

Table 59: Half Yearly Sales Analysis: 2004-2005 (In US$ million)

Table 60: Annual Sales Analysis: 2003-2004 (In US$ million)

74. Osiris Therapeutics, Inc. (USA)

75. Oxford BioMedica PLC (UK)

76. Oxxon Therapeutics Ltd. (UK)

77. Qugen Therapeutics Pte., Ltd. (Singapore)

78. RheoGene, Inc. (USA)

79. Sanofi-Aventis (France)

Table 61: Half Yearly Sales Analysis: 2004-2005 (H1) (In € million)

Table 62: Half Yearly Sales Analysis by Segment: 2004-2005 (H1) (In € million)

Table 63: Half Yearly Sales Analysis by Product: 2004-2005 (H1) (In € million)

Sanofi Aventis Pharmaceuticals, Inc. (USA)

80. Schering AG (Germany)

Berlex Laboratories, Inc. (USA)

Collateral Therapeutics, Inc. (USA)

81. Schering-Plough Corporation (USA)

Table 64: First Quarter Sales Analysis by Segment: 2004-2005 (Q1) (US$ million)

Table 65: First Quarter Sales Analysis by Regions: 2004-2005 (Q1) (US$ million)

Table 66: Annual Sales Analysis by Segments: 2003-2004 (US$ million)

Table 67: Annual Sales Analysis by Regions: 2003-2004 (US$ million)

Canji, Inc. (USA)

82. Selective Genetics (USA)

83. Shanghai Sunway Biotech Co., Ltd. (China)

84. Shenzhen Sibiono Genetech Co., Ltd. (China)

85. Shire Pharmaceuticals Group PLC (UK)

Table 68: Half Yearly Sales Analysis: 2004-2005 (H1) (In US$ million)

Table 69: Annual Sales Analysis: 2003-2004 (In US$ million)

Table 70: Annual Sales Analysis by Product: 2003-2004 (In US$ million)

86. Sirna Therapeutics, Inc. (USA)

87. Stemcells, Inc. (USA)

Table 71: Half Yearly Sales Analysis: 2004-2005 (H1) (In US$ thousand)

Table 72: Annual Sales Analysis: 2003-2004 (In US$ thousand)

88. Supratek Pharma, Inc. (Canada)

89. Targeted Genetics Corporation (USA)

Table 73: Half-Yearly Sales Analysis: 2004-2005 (H1) (In US$ million)

Table 74: Annual Sales Analysis: 2003-2004 (In US$ million)

90. Theratechnologies, Inc. (Canada)

Table 75: Half Yearly Sales Comparison: 2004-2005 (H1) (In C$ million)

Table 76: Annual Sales Comparison: 2003-2004 (In C$ million)

91. Tosk, Inc. (USA)

92. TransGene SA (France)

Table 77: First Half Yearly Sales Analysis: 2004-2005 (H1) (In € thousands)

Table 78: Annual Sales Analysis 2003-2004 (In € thousands)

TransGene, Inc. (USA)

93. Upstate Group, Inc. (USA)

94. Valentis, Inc. (USA)

Table 79: Annual Sales Comparison: 2004-2005 (In US$ million)

Table 80: Quarterly Sales Comparison: 2004-2005 (In US$ million)

95. Vical, Inc. (USA)

Table 81: Half Yearly Sales Analysis: 2004-2005 (H1) (In US$ million)

Table 82: Quarterly Sales Analysis: 2003-2004 (In US$ million)

Table 83: Annual Sales Analysis: 2003-2004 (In US$ million)

96. VirRx, Inc. (USA)

97. VIRxSYS Corporation (USA)

98. Wyeth (USA)

Abstract

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