Genes are composed of DNA that contains necessary information to produce proteins that are vital for the human body to function optimally. Few gene mutations may disrupt protein production or not develop these proteins at all, causing genetic disorders. Gene therapy works by repairing, dis-activating (“turning-off), or replacing dysfunctional genes that cause disease with the aim of (re)establishing normal function. Gene therapy can be used for drug development because with the right target and approach, it can address the root cause of a severe disease. For certain disorders where known genetic mutations lead to deficient or non-functional protein production, gene therapy can “fix” the underlying defect and/or provide a path to producing the functional protein.

CHAPTER 1 INTRODUCTION

1.1. REPORT DESCRIPTION

1.2. KEY BENEFITS

1.3. KEY MARKET SEGMENTS

1.4. RESEARCH METHODOLOGY

1.4.1. Secondary research

1.4.2. Primary research

1.4.3. Analyst tools and models

CHAPTER 2 EXECUTIVE SUMMARY

2.1. CXO PERSPECTIVE

CHAPTER 3 MARKET OVERVIEW

3.1. MARKET DEFINITION AND SCOPE

3.2. KEY FINDINGS

3.2.1. Top investment pockets

3.2.2. Top winning strategies

3.2.2.1. Top winning strategies, by year, 2015–2017*

3.2.2.2. Top winning strategies, by development, 2015–2017* (%)

3.2.2.3. Top winning strategies, by company, 2015–2017*

3.4. PORTER’S FIVE FORCES ANALYSIS

3.5. MARKET DYNAMICS

3.5.1. Drivers

3.5.1.1. High investment for R&D activities pertaining to gene

therapy

3.5.1.2. Increase in prevalence of cancer

3.5.1.3. Growth in awareness regarding gene therapy

3.5.2. Restraints

3.5.2.1. High cost associated with gene therapies

3.5.2.2. Unwanted immune responses

3.5.3. Opportunity

3.5.3.1. Untapped potential for emerging markets

CHAPTER 4 GENE THERAPY MARKET, BY VECTOR

4.1. OVERVIEW

4.1.1. Market size and forecast

4.2. VIRAL VECTORS

4.2.1. Key market trends

4.2.2. Key growth factors and opportunities

4.2.3. Market size and forecast

4.2.4. Market size and forecast by country

4.2.5. Adenovirus

4.2.6. Lentivirus

4.2.7. Retrovirus

4.2.8. Adeno-associated virus

4.2.9. Herpes simplex virus

4.2.10. Poxvirus

4.2.11. Vaccinia virus

4.2.12. Others

4.3. NONVIRAL TECHNIQUES

4.3.1. Key market trends

4.3.2. Key growth factors and opportunities

4.3.3. Market size and forecast

4.3.4. Market size and forecast by country

4.3.5. Naked plasmid vector

4.3.6. Gene gun

4.3.7. Electroporation

4.3.8. Lipofection

4.3.9. Others

CHAPTER 5 GENE THERAPY MARKET, BY GENE

5.1. OVERVIEW

5.1.1. Market size and forecast

5.2. ANTIGEN

5.2.1. Market size and forecast

5.2.2. Market size and forecast by country

5.3. CYTOKINE

5.3.1. Market size and forecast

5.3.2. Market size and forecast, by country

5.4. TUMOR SUPPRESSOR

5.4.1. Market size and forecast

5.4.2. Market size and forecast, by country

5.5. SUICIDE

5.5.1. Market size and forecast

5.5.2. Market size and forecast, by country

5.6. DEFICIENCY

5.6.1. Market size and forecast

5.6.2. Market size and forecast, by country

5.7. GROWTH FACTORS

5.7.1. Market size and forecast

5.7.2. Market size and forecast, by country

5.8. RECEPTORS

5.8.1. Market size and forecast

5.8.2. Market size and forecast, by country

5.9. OTHERS

5.9.1. Market size and forecast

5.9.2. Market size and forecast, by country

CHAPTER 6 GENE THERAPY MARKET, BY APPLICATION

6.1. OVERVIEW

6.1.1. Market size and forecast

6.2. ONCOLOGICAL DISORDERS

6.2.1. Market size and forecast

6.2.2. Market size and forecast, by country

6.3. RARE DISEASES

6.3.1. Market size and forecast

6.3.2. Market size and forecast, by country

6.4. CARDIOVASCULAR DISEASES

6.4.1. Market size and forecast

6.4.2. Market size and forecast, by country

6.5. NEUROLOGICAL DISORDERS

6.5.1. Market size and forecast

6.5.2. Market size and forecast, by country

6.6. INFECTIOUS DISEASES

6.6.1. Market size and forecast

6.6.2. Market size and forecast, by country

6.7. OTHER DISEASES

6.7.1. Market size and forecast

6.7.2. Market size and forecast, by country

CHAPTER 7 GENE THERAPY MARKET, BY REGION

7.1. OVERVIEW

7.1.1. Market size and forecast

7.2. NORTH AMERICA

7.2.1. Key market trends

7.2.2. Key growth factors and opportunities

7.2.3. Market size and forecast, by country

7.2.4. Market size and forecast, by vector type

7.2.5. Market size and forecast, by gene type

7.2.6. Market size and forecast, by application

7.2.6.1. U.S. market size and forecast, by vector type

7.2.6.2. U.S. market size and forecast, by gene type

7.2.6.3. U.S. market size and forecast, by application

7.2.6.4. Canada market size and forecast, by vector type

7.2.6.5. Canada market size and forecast, by gene type

7.2.6.6. Canada market size and forecast, by application

7.2.6.7. Mexico market size and forecast, by vector type

7.2.6.8. Mexico market size and forecast, by gene type

7.2.6.9. Mexico market size and forecast, by application

7.3. EUROPE

7.3.1. Key market trends

7.3.2. Key growth factors and opportunities

7.3.3. Market size and forecast, by country

7.3.4. Market size and forecast, by vector type

7.3.5. Market size and forecast, by gene type

7.3.6. Market size and forecast, by application

7.3.6.1. Germany market size and forecast, by vector type

7.3.6.2. Germany market size and forecast, by gene type

7.3.6.3. Germany market size and forecast, by application

7.3.6.4. France market size and forecast, by vector type

7.3.6.5. France market size and forecast, by gene type

7.3.6.6. France market size and forecast, by application

7.3.6.7. UK market size and forecast, by vector type

7.3.6.8. UK market size and forecast, by gene type

7.3.6.9. UK market size and forecast, by application

7.3.6.10. Italy market size and forecast, by vector type

7.3.6.11. Italy market size and forecast, by gene type

7.3.6.12. Italy market size and forecast, by application

7.3.6.13. Spain market size and forecast, by vector type

7.3.6.14. Spain market size and forecast, by gene type

7.3.6.15. Spain market size and forecast, by application

7.3.6.16. Rest of Europe market size and forecast, by vector type

7.3.6.17. Rest of Europe market size and forecast, by gene type

7.3.6.18. Rest of Europe market size and forecast, by application

7.4. ASIA-PACIFIC

7.4.1. Key market trends

7.4.2. Key growth factors and opportunities

7.4.3. Market size and forecast, by country

7.4.4. Market size and forecast, by vector type

7.4.5. Market size and forecast, by gene type

7.4.6. Market size and forecast, by application

7.4.6.1. Japan market size and forecast, by vector type

7.4.6.2. Japan market size and forecast, by gene type

7.4.6.3. Japan market size and forecast, by application

7.4.6.4. China market size and forecast, by vector type

7.4.6.5. China market size and forecast, by gene type

7.4.6.6. China market size and forecast, by application

7.4.6.7. India market size and forecast, by vector type

7.4.6.8. India market size and forecast, by gene type

7.4.6.9. India market size and forecast, by application

7.4.6.10. South Korea market size and forecast, by vector type

7.4.6.11. South Korea market size and forecast, by gene type

7.4.6.12. South Korea market size and forecast, by application

7.4.6.13. Rest of Asia-Pacific market size and forecast, by vector type

7.4.6.14. Rest of Asia-Pacific market size and forecast, by gene type

7.4.6.15. Rest of Asia-Pacific market size and forecast, by application

7.5. LAMEA

7.5.1. Key market trends

7.5.2. Key growth factors and opportunities

7.5.3. Market size and forecast, by country

7.5.4. Market size and forecast, by vector type

7.5.5. Market size and forecast, by gene type

7.5.6. Market size and forecast, by application

7.5.6.1. Brazil market size and forecast, by vector type

7.5.6.2. Brazil market size and forecast, by gene type

7.5.6.3. Brazil market size and forecast, by application

7.5.6.4. South Africa market size and forecast, by vector type

7.5.6.5. South Africa market size and forecast, by gene type

7.5.6.6. South Africa market size and forecast, by application

7.5.6.7. Rest of LAMEA market size and forecast, by vector type

7.5.6.8. Rest of LAMEA market size and forecast, by gene type

7.5.6.9. Rest of LAMEA market size and forecast, by application

CHAPTER 8 COMPANY PROFILE

8.1. ADAPTIMMUNE THERAPEUTICS PLC.

8.1.1. Company overview

8.1.2. Company snapshot

8.1.3. Operating business segments

8.1.4. Product portfolio

8.1.5. Business performance

8.1.6. Key strategic moves and developments

8.2. BIOCANCELL LTD.

8.2.1. Company overview

8.2.2. Company snapshot

8.2.3. Operating business segments

8.2.4. Product portfolio

8.3. BLUEBIRD BIO, INC.

8.3.1. Company overview

8.3.2. Company snapshot

8.3.3. Operating business segments

8.3.4. Business performance

8.3.5. Key strategic moves and developments

8.4. CELGENE CORPORATION

8.4.1. Company overview

8.4.2. Company snapshot

8.4.3. Operating business segments

8.4.4. Business performance

8.4.5. Key strategic moves and developments

8.5. GILEAD SCIENCES, INC.

8.5.1. Company overview

8.5.2. Company snapshot

8.5.3. Operating business segments

8.5.4. Product portfolio

8.5.5. Business performance

8.5.6. Key strategic moves and developments

8.6. GLAXOSMITHKLINE PLC

8.6.1. Company overview

8.6.2. Company snapshot

8.6.3. Operating business segments

8.6.4. Product portfolio

8.6.5. Business performance

8.6.6. Key strategic moves and developments

8.7. MERCK & CO., INC.

8.7.1. Company overview

8.7.2. Company snapshot

8.7.3. Operating business segments

8.7.4. Product portfolio

8.7.5. Business performance

8.7.6. Key strategic moves and developments

8.8. NOVARTIS AG

8.8.1. Company overview

8.8.2. Company snapshot

8.8.3. Operating business segments

8.8.4. Product portfolio

8.8.5. Business performance

8.8.6. Key strategic moves and developments

8.9. ONCOGENEX PHARMACEUTICALS, INC.

8.9.1. Company overview

8.9.2. Company snapshot

8.9.3. Operating business segments

8.9.4. Business performance

8.9.5. Key strategic moves and developments

8.10. SPARK THERAPEUTICS, INC.

8.10.1. Company overview

8.10.2. Company snapshot

8.10.3. Operating business segments

8.10.4. Product portfolio

8.10.5. Business performance

8.10.6. Key strategic moves and developments

8.11. ABEONA THERAPEUTICS INC.

8.11.1. Company overview

8.11.2. Company snapshot

8.11.3. Operating business segments

8.11.4. Product portfolio

8.11.5. Business performance

8.11.6. Key strategic moves and developments

8.12. ADVERUM BIOTECHNOLOGIES, INC.

8.12.1. Company overview

8.12.2. Company snapshot

8.12.3. Operating business segments

8.12.4. Product portfolio

8.12.5. Key strategic moves and developments

8.13. APPLIED GENETIC TECHNOLOGIES CORPORATION

8.13.1. Company overview

8.13.2. Company snapshot

8.13.3. Operating business segments

8.13.4. Product portfolio

8.13.5. Key strategic moves and developments

8.14. ARBUTUS BIOPHARMA CORPORATION

8.14.1. Company overview

8.14.2. Company snapshot

8.14.3. Product portfolio

8.14.4. Business performance

8.14.5. Key strategic moves and developments

8.15. AUDENTES THERAPEUTICS INC.

8.15.1. Company overview

8.15.2. ompany snapshot

8.15.3. Product portfolio

8.15.4. Key strategic moves and developments

8.16. AVEXIS INC.

8.16.1. Company overview

8.16.2. Company snapshot

8.16.3. Product portfolio

8.16.4. Key strategic moves and developments

8.17. BLUEBIRD BIO, INC.

8.17.1. Company overview

8.17.2. Company snapshot

8.17.3. Operating business segments

8.17.4. Product portfolio

8.17.5. Business performance

8.17.6. Key strategic moves and developments

8.18. CRISPR THERAPEUTICS AG

8.18.1. Company overview

8.18.2. Company snapshot

8.18.3. Product portfolio

8.18.4. Key strategic moves and developments

8.19. EDITAS MEDICINE, INC.

8.19.1. Company overview

8.19.2. Company snapshot

8.19.3. Product portfolio

8.20. INTELLIA THERAPEUTICS INC.

8.20.1. Company overview

8.20.2. Company snapshot

8.20.3. Product portfolio

8.20.4. Business performance

8.21. GILEAD SCIENCES, INC. (KITE PHARMA, INC.)

8.21.1. Company overview

8.21.2. Company snapshot

8.21.3. Operating business segments

8.21.4. Product portfolio

8.21.5. Business performance

8.21.6. Key strategic moves and developments

8.22. REGENXBIO, INC.

8.22.1. Company overview

8.22.2. Company snapshot

8.22.3. Product portfolio

8.22.4. Key strategic moves and developments

8.23. SANGAMO THERAPEUTICS, INC.

8.23.1. Company overview

8.23.2. Company snapshot

8.23.3. Operating business segments

8.23.4. Product portfolio

8.23.5. Business performance

8.23.6. Key strategic moves and developments

8.24. UNIQURE N. V.

8.24.1. Company overview

8.24.2. Company snapshot

8.24.3. Operation segments

8.24.4. Product portfolio

8.24.5. Business performance

8.24.6. Key strategic moves and developments

8.25. VOYAGER THERAPEUTICS, INC.

8.25.1. Company overview

8.25.2. Company snapshot

8.25.3. Operating business segments

8.25.4. Product portfolio

8.25.5. Key strategic moves and developments