Roots Analysis has done a detailed study on Gene Therapy Market (3rd Edition), 2019-2030, covering key aspects of the industry’s evolution and identifying potential future growth opportunities.

Key Market Insights
• In the past four years, more than 31,000 patents related to gene therapies and gene editing have been filed / granted; this is indicative of the heightened pace of research in this domain
• Presently, there are more than 10 approved gene therapies; over 465 product candidates are being evaluated for the treatment of a variety of disease indications
• Most of the therapeutic leads are in the early stages of clinical development; a variety of viral and non-viral vectors are being used to introduce different types of gene modifications in such therapies
• Although start-ups and mid-sized companies are spearheading the innovation, several big biopharmaceutical companies are also actively engaged
• With multiple approved products and several under development, price is one of the major concerns in this market; the future is likely to witness the establishment of more affordable pricing and reimbursement strategies
• As several candidates progress towards approval, developers are exploring diverse commercialization strategies to be implemented across different stages of a product’s launch cycle
• CMOs offering vector manufacturing services have become an integral part of the gene therapy supply chain, owing to their ability to overcome the various associated challenges
• Several investors, having realized the untapped opportunity within this emerging segment of genetic disorders, have invested over USD 16.5 billion across 280 instances, in the period between 2014 and 2019
• Overall, prevalent trends indicate that the market for gene therapies is poised to grow significantly as multiple late stage molecules get commercialized in the near future for the treatment of different therapeutic areas
• The projected future opportunity is expected to be distributed across different types of gene modifications, therapy delivery routes and key geographical regions

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Table of Contents
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 Therapy Development
3.9.2. Concerns Related to Manufacturing
3.9.3. 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
4.2.1.1.1. Overview
4.2.1.1.2. Design
4.2.1.1.3. Advantages
4.2.1.1.4. Limitations
4.2.1.2. Adenoviral Vectors
4.2.1.2.1. Overview
4.2.1.2.2. Design
4.2.1.2.3. Advantages
4.2.1.2.4. Limitations
4.2.1.3. Lentiviral Vectors
4.2.1.3.1. Overview
4.2.1.3.2. Design
4.2.1.3.3. Advantages
4.2.1.3.4. Limitations
4.2.1.4. Retroviral Vectors
4.2.1.4.1. Overview
4.2.1.4.2. Design
4.2.1.4.3. Advantages
4.2.1.4.4. Limitations
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
5.2.1. The US Scenario
5.2.2. The Canadian Scenario
5.3. Regulatory Guidelines in Europe
5.4. Regulatory Guidelines in Asia Pacific
5.4.1. Chinese Scenario
5.4.2. Japanese Scenario
5.4.3. South Korean Scenario
5.4.4. Australian Scenario
5.5. Reimbursement Scenario
5.5.1. Challenges Related to Reimbursement
5.6. Payment Models for Gene Therapies

6. COMPETITIVE LANDSCAPE
6.1. Chapter Overview
6.2. Gene Therapy Market: Clinical and Commercial 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. Gene Therapy: Special Designation Awarded
6.4.1. Analysis by Special Designation Awarded
6.5. Key Players: Analysis by Number of Product Candidates
6.6. Developer Landscape
6.6.1. Distribution by Year of Establishment
6.6.2. Distribution by Size of Developer
6.6.3. Distribution by Geographical Location
6.7. Regional Landscape

7. MARKETED GENE THERAPIES
7.1. Chapter Overview
7.2. Gendicine® (Shenzhen Sibiono GeneTech)
7.2.1. Company Overview
7.2.2. Development Timeline
7.2.3. Mechanism of Action and Vectors Used
7.2.4. Target Indication(s)
7.2.5. Current Status of Development
7.2.6. Manufacturing, Dosage and Sales
7.3. Oncorine® (Shanghai Sunway Biotech)
7.3.1. Company Overview
7.3.2. Development Timeline
7.3.3. Mechanism of Action and Vectors Used
7.3.4. Target Indication(s)
7.3.5. Current Status of Development
7.3.6. Manufacturing, Dosage and Sales
7.4. Rexin-G® (Epeius Biotechnologies)
7.4.1. Company Overview
7.4.2. Development Timeline
7.4.3. Mechanism of Action and Vector Used
7.4.4. Target Indication(s)
7.4.5. Current Status of Development
7.4.6. Manufacturing, Dosage and Sales
7.5. Neovasculgen® (Human Stem Cells Institute)
7.5.1. Company Overview
7.5.2. Development Timeline
7.5.3. Mechanism of Action and Vectors Used
7.5.4. Target Indication(s)
7.5.5. Current Status of Development
7.5.6. Manufacturing, Dosage and Sales
7.6. Imlygic® (Amgen)
7.6.1. Company Overview
7.6.2. Development Timeline
7.6.3. Mechanism of Action and Vectors Used
7.6.4. Target Indication(s)
7.6.5. Current Status of Development
7.6.6. Manufacturing, Dosage and Sales
7.7. Strimvelis® (Orchard Therapeutics)
7.7.1. Company Overview
7.7.2. Development Timeline
7.7.3. Mechanism of Action and Vectors Used
7.7.4. Target Indication(s)
7.7.5. Current Status of Development
7.7.6. Manufacturing, Dosage and Sales
7.8. Invossa™ (Kolon TissueGene)
7.8.1. Company Overview
7.8.2. Development Timeline
7.8.3. Mechanism of Action and Vectors Used
7.8.4. Target Indication(s)
7.8.5. Current Status of Development
7.8.6. Manufacturing, Dosage and Sales
7.9. Luxturna™ (Spark Therapeutics)
7.9.1. Company Overview
7.9.2. Development Timeline
7.9.3. Mechanism of Action and Vector Used
7.9.4. Target Indication(s)
7.9.5. Current Status of Development
7.9.6. Manufacturing, Dosage and Sales
7.10. Zolgensma™ (AveXis / Novartis)
7.10.1. Company Overview
7.10.2. Development Timeline
7.10.3. Mechanism of Action and Vector Used
7.10.4. Target Indication(s)
7.10.5. Current Status of Development
7.10.6. Manufacturing, Dosage and Sales
7.11. Collategene® / Beperminogene Perplasmid (AnGes)
7.11.1. Company Overview
7.11.2. Development Timeline
7.11.3. Mechanism of Action and Vector Used
7.11.4. Target Indication(s)
7.11.5. Current Status of Development
7.11.6. Manufacturing, Dosage and Sales

7.12. Zyntelgo™ (bluebird bio)
7.12.1. Company Overview
7.12.2. Development Timeline
7.12.3. Mechanism of Action and Vector Used
7.12.4. Target Indication(s)
7.12.5. Current Status of Development
7.12.6. Manufacturing, Dosage and Sales

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. AMT-061: Overview of Therapy, Current Development Status and Clinical Results
9.3. BIIB111 (NSR-REP1): Overview of Therapy, Current Development Status and Clinical Results
9.4. BIIB112 (NSR-RPGR): Overview of Therapy, Current Development Status and Clinical Results
9.5. BMN 270 (valoctocogene roxaparvovec): Overview of Therapy, Current Development Status and Clinical Results
9.6. E10A: Overview of Therapy, Current Development Status and Clinical Results
9.7. FLT180a: Overview of Therapy, Current Development Status and Clinical Results
9.8. GS010: Overview of Therapy, Current Development Status and Clinical Results
9.9. Instiladrin®: Overview of Therapy, Current Development Status and Clinical Results
9.10. Lenti-D™: Overview of Therapy, Current Development Status and Clinical Results
9.11. LYS-SAF302: Overview of Therapy, Current Development Status and Clinical Results
9.12. OTL-101: Overview of Therapy, Current Development Status and Clinical Results
9.13. OTL-103: Overview of Therapy, Current Development Status and Clinical Results
9.14. OTL-200: Overview of Therapy, Current Development Status and Clinical Results
9.15. Pexa-Vec (pexastimogene devacirepvec): Overview of Therapy, Current Development Status and Clinical Results
9.16. PF-06838435 (fidanacogene elaparvovec): Overview of Therapy, Current Development Status and Clinical Results
9.17. ProstAtak®: Overview of Therapy, Current Development Status and Clinical Results
9.18. SPK-8011: Overview of Therapy, Current Development Status and Clinical Results
9.19. Toca 511 (vocimagene amiretrorepvec): Overview of Therapy, Current Development Status and Clinical Results
9.20. VB-111 (ofranergene obadenovec): Overview of Therapy, Current Development Status and Clinical Results
9.21. VGX-3100: Overview of Therapy, Current Development Status and Clinical Results
9.22. Vigil®: Overview of Therapy, Current Development Status and Clinical Results
9.23. VM202 (donaperminogene seltoplasmid): 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
11.2. Autoimmune Disorders
11.3.1. Analysis by Target Indication
11.3.2. Analysis by Type of Vector Used
11.3. Cardiovascular Diseases
11.4.1. Analysis by Target Indication
11.4.2. Analysis by Type of Vector Used
11.4. Dermatological Disorders
11.5.1. Analysis by Target Indication
11.5.2. Analysis by Type of Vector Used
11.5. Genetic Disorders
11.6.1. Analysis by Target Indication
11.6.2. Analysis by Type of Vector Used
11.6. Hematological Disorders
11.7.1. Analysis by Target Indication
11.7.2. Analysis by Type of Vector Used
11.7. Infectious Diseases
11.8.1. Analysis by Target Indication
11.8.2. Analysis by Type of Vector Used
11.8. Metabolic Disorders
11.9.1. Analysis by Target Indication
11.9.2. Analysis by Type of Vector Used
11.9. Muscle-related Diseases
11.10.1. Analysis by Target Indication
11.10.2. Analysis by Type of Vector Used
11.10. Nervous System Disorders
11.11.1. Analysis by Target Indication
11.11.2. Analysis by Type of Vector Used
11.11. Oncological Disorders
11.12.1. Analysis by Target Indication
11.12.2. Analysis by Type of Vector Used
11.12. Ophthalmic Diseases
11.13.1. Analysis by Target Indication
11.13.2. Analysis by Type of Vector Used

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
12.3. Gene Editing-related Patents
12.3.1. Scope and Methodology
12.3.1.1. Analysis by Publication Year
12.3.1.2. Analysis by Geographical Location
12.3.1.3. Analysis by CPC Classification
12.3.1.4. Emerging Focus Areas
12.3.1.5. Leading Players: Analysis by Number of Patents
12.3.1.6. Patent Benchmark Analysis
12.3.1.7. Patent Valuation Analysis
12.4. 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
15.1. Chapter Overview
15.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies
15.3. Gene Therapy Market: Pricing Models
15.3.1. On the Basis of Associated Product / Component Costs
15.3.2. On the Basis of Competition
15.3.3. On the Basis of Patient Segment
15.3.4. 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

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