Global & US Gene Therapy Market Forecast to 2020

Published: Feb, 2018 | Pages: 205 | Publisher: RNCOS
Industry: Healthcare | Report Format: Electronic (PDF)

Global & US Gene Therapy Market is expected to grow at a significant CAGR in the upcoming period as the scope and its applications are rising enormously across the globe. Gene Therapy is a technique designed to introduce genetic material into cells to compensate for abnormal genes or to make a useful protein to prevent or treat genetic disease. It is also called as human gene transfer technique.

The factors that are playing a major role in the growth of Gene Therapy Market are growing occurrence of life-threatening and chronic disorders, increasing concentration to accelerate commercialization of gene therapy in developed nations. However, the high cost of gene therapy and strict regulatory laws and safety concerns are the factors restraining overall market growth in the years to come. Gene Therapy Market is segmented based on vectors type, approved gene therapy, application, and region.

Non-viral vectors and viral vectors are the vector’s type that could be explored in Gene Therapy in the forecast period. Non-viral vectors include a gene-activated matrix, naked DNA/plasmid vectors, liposomes, lipoplexes and polyplexes, and oligonucleotides. On the other hand, viral vectors comprise lentiviral vectors, adenoviral vectors, retroviral vectors, and adeno-associated virus vectors.

Based on approval of Gene Therapy is segmented into Gendicine, Rexin-G, Oncorine, Neovasculgen, and Glybera. The market may be categorized based on applications like genetic diseases, oncology, neurological diseases, cardiovascular diseases, infectious diseases, and others could be explored in the forecast period. Gene Therapy Market is segmented geographically into Americas (North America, South America), Europe (Eastern Europe, Western Europe), Asia Pacific (India, China, Japan, New Zealand, and Australia), Middle East (UAE, Saudi, and Qatar) and Africa (South Africa).

Globally, North America may account for the significant market share of Gene Therapy and is estimated to lead the overall market in the years to come. The United States is projected to lead the overall market in terms of revenue. The reason behind the overall market growth could be the presence of key manufacturers in this region. Instead, Europe and the Asia Pacific are also estimated to have a positive influence on the future growth. Europe is the second largest region with significant market share. However, Asia Pacific is estimated to grow at the highest CAGR in the forecast period.

The key players of Gene Therapy Market are Sibiono GeneTech Co. Ltd., Spark Therapeutics, LLC, Shanghai Sunway Biotech Co. Ltd., ViroMed Co. Ltd. dba VM BioPharma, Human Stem Cells Institute, Advantagene Inc., UniQure N.V., Bluebird Bio, Genethon, Sanofi, Oxford BioMedica Plc, and Vical Inc. These players are concentrating on inorganic growth to sustain themselves amongst fierce competition.

Gene therapy is one of the most widely researched fields in the healthcare industry. The high potential of gene therapy to cure various chronic diseases makes it a popular research area. Various researches are being performed across the globe to explore the potential of gene therapy for the treatment of incurable diseases, such as diabetes, cancer, and HIV amongst others.

According to RNCOS’ new research report "Global & US Gene Therapy Market Forecast to 2020”, most of the gene therapy researches are being focused on finding the treatment for cancer, followed by genetic diseases and neurological disorders, respectively. In this context, the gene therapy application chapter of the report provides a comprehensive overview of various diseases in which the gene therapy is used, along with the current and future market size of gene therapy for particular disease and its geographical break up. Primarily, the gene therapy market is dominated by oncology applications, with several companies and academic institutions focusing on novel and ‘difficult to treat’ cancers. Other therapeutic areas seeking developments in gene therapy include monogenic diseases, cardiovascular diseases, infectious diseases, inherited blindness and neurological diseases. Furthermore, the chapter also provides details regarding the various aspects of the clinical trials in the different gene therapy application by phases, vector types, etc.

The major part of the revenue of gene therapy market is generated from research phase. Most of the gene therapy products are in research phases, only few products have been commercialized till date. The report also provides the sales of major marketed gene therapy products, and the list of the products in clinical/pre-clinical research along with their clinical phases.

The companies operating in the gene therapy market are also receiving various funding, grants, and investment from government bodies and venture capitalist firms which are aiding them to develop new products. The study highlights the applications for which the investments have been received.

North America continues to have the maximum number of clinical trials in the gene therapy segment. This is a major reason for the dominant position of North America in the gene therapy market. Based on the geography, the market is divided into four regions, namely, North America, Europe, Asia, and Rest of the World. The report provides the market for each of the geography, along with its forecast till 2020.

RNCOS, in its report, further covers how major trends and drivers, mainly gene silencing, advanced therapies combining gene therapy and stem cell technology, immunodeficiency syndromes, growing interest of venture capital firms, etc. will propel the industry’s growth. An analysis has also been done of a few factors limiting the growth of the industry. The report also provides insights regarding the strategies adopted by the players from 2013 to 2015 for enhancing their market share. Finally, with a view to understand the competitive landscape, the profiles of key market players have been included in the report to present a complete picture of the global gene therapy market.

 Table of Contents

1. Analyst View

2. Research Methodology 

3. Gene Therapy - An Introduction
     3.1 Classification of Gene Therapy Techniques
     3.2 Physical Methods of Gene Transfer
           3.2.1 Electroporation
           3.2.2 Hydrodynamic
           3.2.3 Microinjection
           3.2.4 Particle Bombardment
           3.2.5 Ultrasound-Mediated Transfection
     3.3 Vectors for Gene Therapy
           3.3.1 Viral Vectors
                    3.3.1.1 Adenoviral Vectors
                    3.3.1.2 Adeno-associated Virus Vectors
                    3.3.1.3 Retroviral Vectors
                    3.3.1.4 Lentiviral Vectors
           3.3.2 Non Viral Vectors
                   3.3.2.1 Naked DNA/Plasmid Vectors
                   3.3.2.2 Oligonucleotides
                   3.3.2.3 Liposomes, Lipoplexes and Polyplexes
                   3.3.2.4 Gene-Activated Matrix

4. Industry Overview
    4.1 Market Drivers
         4.1.1 Failure of Conventional Therapies to Treat Cancers
         4.1.2 Rising Focus to Accelerate Commercialization of Gene Therapy in Developed Nations 
         4.1.3 Rising Incidence of Chronic and Life-Threatening Diseases
         4.1.4 Growing Interest of Venture Capital Firms
    4.2 Market Restraints
         4.2.1 Stringent Regulatory Laws & Safety Concerns 
         4.2.2 Challenges in Commercialization of Gene Therapy
         4.2.3 High Cost of the Gene Therapy Drugs
         4.2.4 Pitfalls in Current Technique
         4.2.5 Production & Manufacturing: A Unique Challenge for Gene Therapies 
    4.3 Market Opportunities
         4.3.1 Approval of Gene Therapy Drug in Europe
         4.3.2 Gene Therapy for Hemophilia B Offers Significant Opportunity 
    4.4 Industry Trends
         4.4.1 Gene Silencing: Gaining Momentum
         4.4.2 Advanced Therapies: Combining Gene Therapy and Stem Cell Technology
         4.4.3 Immunodeficiency Syndromes: The Focus of Gene Therapy
         4.4.4 Nanotechnology: Empowering Gene Therapy
         4.4.5 Gene Therapy: A New Hope to Treat Blindness
         4.4.6 Gene Therapy: Potential Cure for Cancer
    4.5 Winning Imperatives
          4.5.1 Investment in R&D Activities

5. Clinical Trial Assessment & Pipeline Analysis
    5.1 Clinical Trials
         5.1.1 By Geography
          5.1.2 By Indication
              5.1.3 By Gene Type
              5.1.4 By Vector
              5.1.5 By Clinical Trial Phase
      5.2 Pipeline Analysis

6. Gene Therapy Market - Regulatory Landscape & Reimbursement Scenario
    6.1 Regulatory Landscape
          6.1.1 US
            6.1.2 Canada
            6.1.3 Europe 
            6.1.4 Japan
            6.1.5 China
            6.1.6 India
            6.1.7 Australia
     6.2 Reimbursement Scenario in Global Market
7. Marketed Gene Therapies
     7.1 Neovasculgen
     7.2 Glybera
      7.3 Gendicine, Rexin-G, Oncorine
              7.3.1 Gendicine
              7.3.2 Rexin G 
              7.3.3 Oncorine

8. Gene Therapy Market
     8.1 Global
     8.2 US

9. Gene Therapy Market by Application
     9.1 Oncology
             9.1.1 Clinical Trials 
             9.1.2 Prevalence
             9.1.3 Market Outlook
     9.2 Cardiovascular Diseases
             9.2.1 Clinical Trials
             9.2.2 Prevalence
             9.2.3 Market Outlook
      9.3 Infectious Diseases
             9.3.1 Clinical Trials
             9.3.2 Prevalence
             9.3.3 Market Outlook
       9.4 Neurological Diseases
              9.4.1 Clinical Trials
              9.4.2 Prevalence
              9.4.3 Market Outlook
       9.5 Genetic Diseases
              9.5.1 Clinical Trials
             9.5.2 Prevalence
             9.5.3 Market Outlook
       9.6 Others 
               9.6.1 Clinical Trials
              9.6.2 Prevalence
             9.6.3 Market Outlook

10. Gene Therapy Market Size by Geography
       10.1 North America
       10.2 Europe
       10.3 Asia
       10.4 Rest of the World (RoW)

11. Competitive Landscape
       11.1 Strategies Adopted by Various Players
                 11.1.1 Strategic Collaborations
                 11.1.2 Acquisitions 
                 11.1.3 Funding & Investments
       11.2 Companies Progress in Gene Therapy In 2015 

12. Key Players Analysis (Business Description, Clinical Pipeline, Recent Developments, Strengths & Weaknesses)
       12.1 Spark Therapeutics, LLC
       12.2 ViroMed Co. Ltd. dba VM BioPharma
       12.3 Advantagene Inc.
       12.4 Bluebird Bio
       12.5 Sanofi
       12.6 Vical Inc.
       12.7 Oxford BioMedica Plc
       12.8 Genethon
       12.9 uniQure N.V.
       12.10 Human Stem Cells Institute
       12.11 Shanghai Sunway Biotech Co. Ltd.
       12.12 Sibiono GeneTech Co. Ltd.

List of Tables:

Table 4-1: US – Promising Gene Therapies in Late-Stage Development
Table 4-2: Europe - Promising Gene Therapies in Late-Stage Development
Table 4-3: Gene Therapy Companies Funded by Venture Capital Firms
Table 4-4: Current Status of Clinical Trials for Hemophilia
Table 5-1: Human Gene Transfer Trials Approved by Condition
Table 5-2: Global - Key Gene Therapy Ongoing Clinical Trials
Table 5-3: US - Key Gene Therapy Ongoing Clinical Trials
Table 6-1: China - Key Players and their Products in Gene Therapy
Table 9-1: Global - Key Oncology Gene Therapy Ongoing Clinical Trials
Table 9-2: US - Key Oncology Gene Therapy Ongoing Clinical Trials
Table 9-3: Global - Oncology Incidence by Geography (‘000), 2015 & 2020
Table 9-4: US - Oncology Incidence (‘000), 2015 & 2020
Table 9-5: Global - Key Infectious Disease Gene Therapy Ongoing Clinical Trials
Table 9-6: US - Key Infectious Disease Gene Therapy Ongoing Clinical Trials
Table 9-7: Global - HIV Prevalence & Incidence by Geography (‘000), 2014
Table 9-8: Global - Key Neurological Disease Gene Therapy Ongoing Clinical Trials
Table 9-9: US - Key Neurological Disease Gene Therapy Ongoing Clinical Trials
Table 9-10: Global - Parkinson Disease Prevalence by Geography (Million), 2010
Table 9-11: US - Parkinson Disease Prevalence (Million), 2010
Table 9-12: Global - Key Genetic Disease Gene Therapy Ongoing Clinical Trials
Table 9-13: US - Key Genetic Disease Gene Therapy Ongoing Clinical Trials
Table 9-14: Global - Hemophilia Prevalence by Geography, 2010 & 2013 
Table 9-15: US - Hemophilia Prevalence, 2010 & 2013
Table 9-16: Global - Key Other Disease Gene Therapy Ongoing Clinical Trials
Table 9-17: US - Key Other Disease Gene Therapy Ongoing Clinical Trials
Table 12-1: Spark Therapeutics LLC - Clinical Pipeline in Gene Therapy
Table 12-2: ViroMed Co. Ltd. - Clinical Pipeline in Gene Therapy
Table 12-3: Advantagene Inc. - Clinical Pipeline in Gene Therapy
Table 12-4: Bluebird Bio - Clinical Pipeline in Gene Therapy
Table 12-5: Sanofi - Clinical Pipeline in Gene Therapy
Table 12-6: Vical Inc. - Clinical Pipeline in Gene Therapy
Table 12-7: Oxford Biomedica Plc – Clinical Pipeline in Gene Therapy 
Table 12-8: Genethon - Clinical Pipeline in Gene Therapy
Table 12-9: UniQure N.V. - Clinical Pipeline in Gene Therapy
Table 12-10: Human Stem Cells Institute - Clinical Pipeline in Gene Therapy
Table 12-11: Shanghai Sunway Biotech Co. Ltd. - Clinical Pipeline in Gene Therapy
Table 12-12: Sibiono GeneTech Co. Ltd. - Clinical Pipeline in Gene Therapy

List of Figures:

Figure 4-1: Global - Major Venture Capitalist Investments in Gene Therapy Product Development (Billion US$), 1999 to 2015
Figure 5-1: Global - Number of Approved Gene Therapy Clinical Trials (2005-2015)
Figure 5-2: Global - Geographical Distribution of Gene Therapy Clinical Trials by Continents (%)
Figure 5-3: Global - Geographical Distribution of Gene Therapy Clinical Trials by Countries (%)
Figure 5-4: US - Geographical Distribution of Gene Therapy Clinical Trials by States (%)
Figure 5-5: Global - Gene Therapy Clinical Trials by Indication (%)
Figure 5-6: US - Gene Therapy Clinical Trials by Indication (%)
Figure 5-7: Global - Gene Therapy Clinical Trials by Types of Gene Transferred (%)
Figure 5-8: Global - Gene Therapy Clinical Trials by Vectors (%)
Figure 5-9: US - Gene Therapy Clinical Trials by Vectors (%)
Figure 5-10: Global - Gene Therapy Clinical Trials by Phase (%)
Figure 5-11: US - Gene Therapy Clinical Trials by Phase (%)
Figure 6-1: US - Number of New Cell Therapy and Gene Therapy Products Submission to FDA (2003-2010)
Figure 6-2: Japan - Evaluation System of Gene Therapy Clinical Trial
Figure 7-1: Global - Neovasculgen Sales (Million US$), 2013 & 2014
Figure 8-1: Global - Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 8-2: US - Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 9-1: Global - Gene Therapy Market by Application (%), 2015
Figure 9-2: Global - Gene Therapy Market by Application (%), 2020
Figure 9-3: US - Gene Therapy Market by Application (%), 2015
Figure 9-4: US - Gene Therapy Market by Application (%), 2020
Figure 9-5: Global - Oncology Gene Therapy Clinical Trials by Vectors (%) 
Figure 9-6: US - Oncology Gene Therapy Clinical Trials by Vectors (%)
Figure 9-7: Global - Oncology Gene Therapy Clinical Trials by Phase (%) 
Figure 9-8: US - Oncology Gene Therapy Clinical Trials by Phase (%)
Figure 9-9: Global - Oncology Gene Therapy Clinical Trials by Type (%)
Figure 9-10: US - Oncology Gene Therapy Clinical Trials by Type (%)
Figure 9-11: Global - Oncology Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 9-13: Global - Oncology Gene Therapy Market by Geography (%), 2015
Figure 9-12: US - Oncology Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 9-14: Global - Key Cardiovascular Diseases Gene Therapy Ongoing Clinical Trials
Figure 9-15: US - Key Cardiovascular Diseases Gene Therapy Ongoing Clinical Trials
Figure 9-16: Global - Cardiovascular Disease Gene Therapy Clinical Trials by Vectors (%)
Figure 9-17: US - Cardiovascular Disease Gene Therapy Clinical Trials by Vectors (%)
Figure 9-18: Global - Cardiovascular Disease Gene Therapy Clinical Trials by Phase (%)
Figure 9-19: US - Cardiovascular Disease Gene Therapy Clinical Trials by Phase (%)
Figure 9-20: Global - Cardiovascular Disease Gene Therapy Clinical Trials by Type (%)
Figure 9-21: US - Cardiovascular Disease Gene Therapy Clinical Trials by Type (%)
Figure 9-22: Global - Cardiovascular Disease Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 9-24: Global - Cardiovascular Disease Gene Therapy Market by Geography (%), 2015
Figure 9-23: US - Cardiovascular Disease Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 9-25: Global - Infectious Disease Gene Therapy Clinical Trials by Vectors (%)
Figure 9-26: US - Infectious Disease Gene Therapy Clinical Trials by Vectors (%)
Figure 9-27: Global - Infectious Disease Gene Therapy Clinical Trials by Phase (%)
Figure 9-28: US - Infectious Disease Gene Therapy Clinical Trials by Phase (%)
Figure 9-29: Global - Infectious Disease Gene Therapy Clinical Trials by Type (%)
Figure 9-30: US - Infectious Disease Gene Therapy Clinical Trials by Type (%)
Figure 9-31: Global - Infectious Disease Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 9-33: Global - Infectious Disease Gene Therapy Market by Geography (%), 2015
Figure 9-32: US - Infectious Disease Gene Therapy Market (Million US$), 2014, 2015 & 2020 
Figure 9-34: Global - Neurological Diseases Gene Therapy Clinical Trials by Vector (%)
Figure 9-35: US - Neurological Diseases Gene Therapy Clinical Trials by Vector (%)
Figure 9-36: Global - Neurological Diseases Gene Therapy Clinical Trials by Phase (%)
Figure 9-37: US - Neurological Diseases Gene Therapy Clinical Trials by Phase (%)
Figure 9-38: Global - Neurological Diseases Gene Therapy Clinical Trials by Type (%) 
Figure 9-39: US - Neurological Diseases Gene Therapy Clinical Trials by Type (%)
Figure 9-40: Global - Neurological Disease Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 9-42: Global - Neurological Disease Gene Therapy Market by Geography (%), 2015
Figure 9-41: US - Neurological Disease Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 9-43: Global - Genetic Disease Gene Therapy Clinical Trials by Vector (%)
Figure 9-44: US - Genetic Disease Gene Therapy Clinical Trials by Vector (%)
Figure 9-45: Global - Genetic Disease Gene Therapy Clinical Trials by Phase (%)
Figure 9-46: US - Genetic Disease Gene Therapy Clinical Trials by Phase (%)
Figure 9-47: Global - Genetic Disease Gene Therapy Clinical Trials by Type (%)
Figure 9-48: US - Genetic Disease Gene Therapy Clinical Trials by Type (%)
Figure 9-49: Global - Genetic Disease Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 9-50: Global - Genetic Disease Gene Therapy Market, by Geography (%), 2015
Figure 9-51: US - Genetic Disease Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 9-52: US – Other Disease Gene Therapy Clinical Trials by Vector (%)
Figure 9-53: US - Other Disease Gene Therapy Clinical Trials by Phase (%)
Figure 9-54: Global - Other Disease Gene Therapy Clinical Trials by Type (%)
Figure 9-55: US - Other Disease Gene Therapy Clinical Trials by Type (%)
Figure 9-56: Global - Other Disease Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 9-57: Global - Other Disease Gene Therapy Market by Geography (%), 2015
Figure 9-58: US - Other Disease Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 10-1: Global - Gene Therapy Market by Geography (%), 2015
Figure 10-2: North America - Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 10-3: Europe - Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 10-4: Asia - Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 10-5: Asia - Number of Gene Therapy Laboratories by Major Asian Countries (2014)
Figure 10-6: India - Number of Gene Therapy Laboratories (2000, 2010 & 2013)
Figure 10-7: Rest of the World - Gene Therapy Market (Million US$), 2014, 2015 & 2020
Figure 11-1: Key Growth Strategies Adopted by Key Players (2013 to 2015)
Figure 11-2: Key Players Focusing on Strategic Collaborations (2013 to 2015)
Figure 11-3: Key Players Focusing on Strategic Acquisitions (2013 to 2015)
Figure 11-4: Key Players Focusing on Funding & Investments (2013 to 2015)
Figure 11-5: Companies Progress in Gene Therapy till 2015