Gene Therapy Market, 2030 - Key Therapeutic Areas, Types of Vectors, Therapy Types, Gene Modification, Regional Analysis

DUBLIN, Sept. 30, 2019 /PRNewswire/ -- The "Gene Therapy Market (3rd Edition), 2019-2030" report has been added to ResearchAndMarkets.com's offering.

The Gene Therapy Market (3rd Edition), 2019-2030 report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, and genome editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

One of the key objectives of the report was to estimate the existing market size and the future opportunity for gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates, and expected pricing, we have provided informed estimates on the evolution of the market for the period 2019-2030.

The report also features the likely distribution of the current and forecasted opportunity across:

• [A] key therapeutic areas (cardiovascular disorders, muscular disorders, neurological disorders, ocular disorders, oncology and others)
• [B] various types of vectors used for therapy development (adeno associated virus, adenovirus, lentivirus, plasmid DNA, retrovirus and others)
• [C] type of therapy (ex vivo and in vivo)
• [D] type of gene modification (gene augmentation, oncolytic viral therapy and others)
• [E] key geographical regions (US, EU5 and rest of the world)

Key Topics Covered:

1 Preface
1.1. Scope of the Report
1.2. Research Methodology
1.3. Chapter Outlines

2 Executive Summary

3 Introduction
3.1. Context and Background
3.2. Evolution of Gene Therapies
3.3. Classification of Gene Therapies
3.3.1. Somatic and Germline Gene Therapy
3.3.2. Ex Vivo and In Vivo Gene Therapy
3.4. Routes of Administration
3.5. Mechanism of Action of Gene Therapies
3.6. Concept of Gene Editing
3.7. Advantages and Disadvantages of Gene Therapies
3.8. Ethical and Social Concerns Related to Gene Therapies
3.9. Future Constraints and Challenges Related to Gene Therapies
3.9.1. Concerns Related to Manufacturing
3.9.2. Concerns Related to Commercial Viability

4 Gene Delivery Vectors
4.1. Chapter Overview
4.2. Viral Vectors
4.2.1 Types of Viral Vectors
4.2.1.1. Adeno-associated Viral Vectors
4.2.1.2. Adenoviral Vectors
4.2.1.3. Lentiviral Vectors
4.2.1.4. Retroviral Vectors
4.2.1.5. Other Viral Vectors
4.2.1.5.1. Alphavirus
4.2.1.5.2. Herpes Simplex Virus
4.2.1.5.3. Simian Virus
4.2.1.5.4. Vaccinia Virus
4.3. Non-Viral Vectors
4.3.1. Types of Non-Viral Vectors
4.3.1.1. Plasmid DNA
4.3.1.2. Liposomes, Lipoplexes and Polyplexes
4.3.1.3. Oligonucleotides
4.4. Methods of Transfection
4.4.1. Biolistic Method
4.4.2. Electroporation
4.4.3. Receptor Mediated Gene Delivery
4.4.4. Gene Activated Matrix (GAM)

5 Regulatory Landscape And Reimbursement Scenario
5.1. Chapter Overview
5.2. Regulatory Guidelines in North America
5.3. Regulatory Guidelines in Europe
5.4. Regulatory Guidelines in Asia Pacific
5.5. Reimbursement Scenario
5.6. Payment Models for Gene Therapies

6 Competitive Landscape
6.1. Chapter Overview
6.2. Gene Therapy Market: Clinical Pipeline
6.2.1. Analysis by Phase of Development
6.2.2. Analysis by Therapeutic Area
6.2.3. Analysis by Type of Vector Used
6.2.4. Analysis by Type of Gene
6.2.5. Analysis by Type of Modification
6.2.6. Analysis by Type of Gene Therapy
6.2.7. Analysis by Route of Administration
6.3. Gene Therapy Market: Early Stage Pipeline
6.3.1. Analysis by Stage of Development
6.3.2. Analysis by Therapeutic Area
6.3.3. Analysis by Type of Vector Used
6.3.4. Analysis by Type of Gene
6.3.5. Analysis by Type of Modification
6.3.6. Analysis by Type of Gene Therapy
6.4. Key Players
6.5. Developer Landscape
6.6. Regional Landscape

7 Marketed Gene Therapies
7.1. Chapter Overview
7.2. Gendicine (Shenzhen Sibiono GeneTech)
7.3. Oncorine (Shanghai Sunway Biotech)
7.4. Rexin-G (Epeius Biotechnologies)
7.5. Neovasculgen (Human Stem Cells Institute)
7.6. Imlygic (Amgen)
7.7. Strimvelis (Orchard Therapeutics)
7.8. Invossa (TissueGene)
7.9. Luxturna (Spark Therapeutics)
7.10. Zolgensma (AveXis / Novartis)
7.11. Collategene / Beperminogene Perplasmid (AnGes)
7.12. Zyntelgo (bluebird bio)

8 Key Commercialization Strategies
8.1. Chapter Overview
8.2. Successful Drug Launch Strategy: ROOTS Framework
8.3. Successful Drug Launch Strategy: Product Differentiation
8.4. Commonly Adopted Commercialization Strategies based on Development Stage of the Product
8.5. Approved Gene Therapies
8.6. Key Commercialization Strategies Adopted by Companies Focused on Gene Therapy
8.6.1. Strategies Adopted Before Therapy Approval
8.6.2. Strategies Adopted During / Post Therapy Approval
8.7. Concluding Remarks

9 Late Stage (Phase Ii/Iii And Above) Gene Therapies
9.1. Chapter Overview
9.2. Axalimogene Filolisbac: Overview of Therapy, Current Development Status and Clinical Results
9.3. AMT-061: Overview of Therapy, Current Development Status and Clinical Results
9.4. BIIB111: Overview of Therapy, Current Development Status and Clinical Results
9.5. BIIB112: Overview of Therapy, Current Development Status and Clinical Results
9.6. Donaperminogene Seltoplasmid (VM202): Overview of Therapy, Current Development Status and Clinical Results
9.7. E10A: Overview of Therapy, Current Development Status and Clinical Results
9.8. Fidanacogene Elaparvovec (PF-06838435): Overview of Therapy, Current Development Status and Clinical Results
9.9. FLT180a: Overview of Therapy, Current Development Status and Clinical Results
9.10. GS010: Overview of Therapy, Current Development Status and Clinical Results
9.11. Instiladrin: Overview of Therapy, Current Development Status and Clinical Results
9.12. Lenti-D: Overview of Therapy, Current Development Status and Clinical Results
9.13. LYS-SAF302: Overview of Therapy, Current Development Status and Clinical Results
9.14. Ofranergene Obadenovec (VB-111): Overview of Therapy, Current Development Status and Clinical Results
9.15. OTL-101: Overview of Therapy, Current Development Status and Clinical Results
9.16. OTL-103: Overview of Therapy, Current Development Status and Clinical Results
9.17. OTL-200: Overview of Therapy, Current Development Status and Clinical Results
9.18. Pexastimogene Devacirepvec (PEXA-VEC): Overview of Therapy, Current Development Status and Clinical Results
9.19. ProstAtak: Overview of Therapy, Current Development Status and Clinical Results
9.20. SPK-8011: Overview of Therapy, Current Development Status and Clinical Results
9.21. Unnamed Therapy: Overview of Therapy, Current Development Status and Clinical Results
9.22. Valoctocogene Roxaparvovec (BMN 270): Overview of Therapy, Current Development Status and Clinical Results
9.23. Vigil: Overview of Therapy, Current Development Status and Clinical Results
9.24. VGX-3100: Overview of Therapy, Current Development Status and Clinical Results
9.25. Vocimagene Amiretrorepvec (Toca-511): Overview of Therapy, Current Development Status and Clinical Results

10 Emerging Technologies
10.1. Chapter Overview
10.2. Gene Editing Technologies
10.2.1. Overview
10.2.2. Applications
10.3. Emerging Gene Editing Platforms
10.3.1. CRISPR / Cas9 System
10.3.2. TALENs
10.3.3. megaTAL
10.3.4. Zinc Finger Nuclease
10.4. Gene Expression Regulation Technologies
10.5. Technology Platforms for Developing / Delivering Gene Therapies

11 Promising Therapeutics Areas
11.1. Chapter Overview
11.2 Analysis by Special Designations Awarded
11.3. Cardiovascular Disorders
11.3.1. Analysis by Target Indication
11.3.2. Analysis by Type of Vector Used
11.4. Hematological Disorders
11.5. Inflammatory & Infectious (I&I) Diseases
11.6. Metabolic Disorders
11.7. Muscular Disorders
11.8. Neurological Disorders
11.9. Ophthalmic Disorders
11.10. Oncology

12 Patent Analysis
12.1. Chapter Overview
12.2. Gene Therapy-related Patents
12.2.1. Scope and Methodology
12.2.1.1. Analysis by Publication Year
12.2.1.2. Analysis by Geographical Location
12.2.1.3. Analysis by CPC Classification
12.2.1.4. Emerging Focus Areas
12.2.1.5. Leading Players: Analysis by Number of Patents
12.2.1.6. Patent Benchmark Analysis
12.2.1.7. Patent Valuation Analysis
12.2. Gene Editing-related Patents
12.2.1. Scope and Methodology
12.2.1.1. Analysis by Publication Year
12.2.1.2. Analysis by Geographical Location
12.2.1.3. Analysis by CPC Classification
12.2.1.4. Emerging Focus Areas
12.2.1.5. Leading Players: Analysis by Number of Patents
12.2.1.6. Patent Benchmark Analysis
12.2.1.7. Patent Valuation Analysis
12.3. Overall Intellectual Property Portfolio: Analysis by Type of Organization

13 Mergers And Acquisitions
13.1. Chapter Overview
13.2. Merger and Acquisition Models
13.3. Gene Therapy: Mergers and Acquisitions
13.3.1. Analysis by Year of Mergers and Acquisitions
13.3.2. Analysis by Type of Mergers and Acquisitions
13.3.3. Regional Analysis
13.3.3.1. Continent-wise Distribution
13.3.3.2. Intercontinental and Intracontinental Deals
13.3.3.3. Country-wise Distribution
13.3.4. Analysis by Key Value Drivers
13.3.4.1. Analysis by Key Value Drivers and Year of Acquisition
13.3.5. Analysis by Phase of Development of the Acquired Company's Product
13.3.6. Analysis by Therapeutic Area

14 Funding And Investment Analysis
14.1. Chapter Overview
14.2. Types of Funding
14.3. Funding and Investment Analysis
14.3.1. Analysis by Number of Funding Instances
14.3.2. Analysis by Amount Invested
14.3.3. Analysis by Type of Funding
14.3.4. Analysis by Amount Invested across Different Types of Therapies
14.3.5. Regional Analysis by Amount Invested
14.3.6. Most Active Players
14.3.7. Key Investors
14.3.8. Analysis by Stage of Development
14.4. Concluding Remarks

15 Cost Price Analysis
13.1. Chapter Overview
13.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies
13.3. Gene Therapy Market: Pricing Models
13.3.1. On the Basis of Associated Product / Component Costs
13.3.2. On the Basis of Competition
13.3.3. On the Basis of Patient Segment
13.3.4. On the Basis of Opinions of Industry Experts

16 Big Pharma Players: Analysis Of Gene Therapy Related Initiatives
16.1. Chapter Overview
16.2. Top Pharmaceutical Companies
16.2.1. Analysis by Therapeutic Area
16.2.2. Analysis by Type of Vector Used
16.2.3. Analysis by Type of Modification
16.2.4. Analysis by Type of Gene Therapy
16.3. Other Big Pharma Players

17 Market Forecast And Opportunity Analysis
17.1. Chapter Overview
17.2. Scope and Limitations
17.3. Key Assumptions and Forecast Methodology
17.4. Overall Gene Therapy Market, 2019-2030
17.4.1. Gene Therapy Market: Analysis by Type of Gene Modification
17.4.2. Gene Therapy Market: Analysis by Type of Therapy
17.4.3. Gene Therapy Market: Analysis by Type of Vector Used
17.4.4. Gene Therapy Market: Analysis by Therapeutic Area
17.4.5. Gene Therapy Market: Analysis by Route of Administration
17.4.6. Gene Therapy Market: Analysis by Geography
17.5. Gene Therapy Market: Value Creation Analysis
17.6. Gene Therapy Market: Product-wise Sales Forecasts
17.6.1. Gendicine
17.6.1.1. Target Patient Population
17.6.1.2. Sales Forecast
17.6.1.3. Net Present Value
17.6.1.4. Value Creation Analysis
17.6.2. Oncorine
17.6.3. Rexin-G
17.6.4. Neovasculgen
17.6.5. Strimvelis
17.6.6. Imlygic
17.6.7. Invossa
17.6.8. Luxturna
17.6.9. Zolgensma
17.6.10. Collategene / Beperminogene Perplasmid
17.6.11. Zyntelgo
17.6.12. Axalimogene Filolisbac
17.6.13. AMT-061
17.6.14. BIIB111
17.6.15. BIIB112
17.6.16. Donaperminogene Seltoplasmid (VM202)
17.6.17. E10A
17.6.18. Fidanacogene Elaparvovec (PF-06838435)
17.6.19. FLT180a
17.6.20. GS010
17.6.21. Instiladrin
17.6.22. Lenti-D
17.6.23. LYS-SAF302
17.6.24. Ofranergene Obadenovec (VB-111)
17.6.25. OTL-101
17.6.26. OTL-103
17.6.27. OTL-200
17.6.28. Pexastimogene Devacirepvec (PEXA-VEC)
17.6.29. ProstAtak
17.6.30. SPK-8011
17.6.31. Unnamed Therapy
17.6.32. Valoctocogene Roxaparvovec (BMN 270)
17.6.33. Vigil
17.6.34. VGX-3100
17.6.35. Vocimagene Amiretrorepvec (Toca-511)

18 Vector Manufacturing
18.1. Chapter Overview
18.2. Overview of Viral Vector Manufacturing
18.3. Viral Vector Manufacturing Processes
18.3.1. Mode of Vector Production
18.3.2. Adherent and Suspension Cultures
18.3.3. Unit Processes and Multiple Parallel Processes
18.3.4. Cell Culture Systems for Production of Viral Vectors
18.3.5. Culture Media Specifications
18.4. Bioprocessing of Viral Vectors
18.4.1. AAV Vector Production
18.4.2. Adenoviral Vector Production
18.4.3. Lentiviral Vector Production
18.4.4. -Retroviral Vector Production
18.5. Challenges Associated with Vector Manufacturing
18.6. Companies Offering Contract Services for Viral and Plasmid Vectors

19 Case Study: Gene Therapy Supply Chain
19.1. Chapter Overview
19.2. Overview of the Gene Therapy Supply Chain
19.3. Implementation of Supply Chain Models
19.4. Logistics in Gene Therapy
19.4.1. Logistics Processes for Autologous and Allogeneic Therapies
19.5. Regulatory Supply Chain across the Globe
19.6. Challenges Associated with Gene Therapy Supply Chain
19.7. Optimizing Cell and Advanced Therapies Supply Chain Management
19.8. Recent Developments and Upcoming Trends

20 Conclusion
20.1. Chapter Overview
20.2. Key Takeaways

21 Interview Transcripts
21.1. Chapter Overview
21.2. Adam Rogers, Chief Executive Officer, Hemera Biosciences
21.3. Al Hawkins, Chief Executive Officer, Milo Biotechnology
21.4. Buel Dan Rodgers, Founder & Chief Executive Officer, AAVogen
21.5. Cedric Szpirer, Executive & Scientific Director, Delphi Genetics
21.6. Christopher Reinhard, Chief Executive Officer and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)
21.7. Jeffrey Hung, Chief Commercial Officer, Vigene Biosciences
21.8. Marco Schmeer, Project Manager and Tatjana Buchholz, Marketing Manager, PlasmidFactory
21.9. Michael Tripletti, Chief Executive Officer, Myonexus Therapeutics
21.10. Robert Jan Lamers, Chief Executive Officer, Arthrogen
21.11. Ryo Kubota, Chairman, President and Chief Executive Officer, Acucela
21.12. Tom Wilton, Chief Business Officer, LogicBio Therapeutics

22 Appendix 1: Tabulated Data

23 Appendix 2: List Of Companies And Organizations

For more information about this report visit https://www.researchandmarkets.com/r/qx40lm

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