Table of Content

1. PREFACE
1.1. Scope of the Report
1.2. Market Segmentation
1.3. Research Methodology
1.4. Key Questions Answered
1.5. Chapter Outlines

2. EXECUTIVE SUMMARY

3. INTRODUCTION
3.1. Chapter Overview
3.2. Viral and Non-Viral Vectors
3.2.1. Viral Vectors
3.2.1.1. Adenovirus Vectors
3.2.1.2. Adeno-Associated Viral Vectors
3.2.1.3. Lentivirus Vectors
3.2.1.4. Retrovirus Vectors
3.2.1.5. Other Viral Vectors
3.2.1.5.1. Alphavirus
3.2.1.5.2. Foamy Virus
3.2.1.5.3. Simian Virus
3.2.1.5.4. Vaccinia Virus
3.2.1.5.5. Chimeric Viral Vectors
3.2.1.5.6. Herpes Simplex Virus
3.2.1.5.7. Sendai Virus
3.2.2. Non-Viral Vectors
3.2.2.1. Plasmid DNA
3.2.2.2. Oligonucleotides
3.2.2.3. Liposomes, Lipoplexes, and Polyplexes
3.2.2.4. Other Non-Viral Vectors
3.3. Adeno-Associated Viral Vectors
3.3.1. Structure and Design
3.3.2. Adeno-Associated Viral Vector Life Cycle
3.3.3. Applications of Adeno-Associated Viral Vectors
3.3.3.1. Gene Therapy
3.3.3.2. Vaccination
3.3.4. Advantages of Adeno-Associated Viral Vectors
3.3.5. Challenges Related to Adeno-Associated Viral Vectors
3.4. Concluding Remarks

4. AAV BASED GENE THERAPY: MARKET LANDSCAPE
4.1. Chapter Overview
4.2. AAV Based Gene Therapy: Overall Market Landscape
4.2.1. Analysis by Phase of Development
4.2.2. Analysis by Therapeutic Area(s)
4.2.3. Analysis by Type of Gene / Molecule Targeted
4.2.4. Analysis by Type of Therapy
4.2.5. Analysis by Type of Gene Delivery Method Used
4.2.6. Analysis by Route of Administration
4.3. AAV Based Gene Therapy Candidates: Special Designations
4.3.1. Analysis by Special Designation(s) Awarded
4.4. AAV Based Gene Therapy: List of Developers
4.4.1. Analysis by Year of Establishment
4.4.2. Analysis by Company Size
4.4.3. Analysis by Location of Headquarters
4.4.4. AAV Based Gene Therapy: Leading Developers

5. AAV MANUFACTURING COMPANIES: MARKET LANDSCAPE
5.1. Chapter Overview
5.2. AAV Manufacturing Companies: Overall Market Landscape
5.2.1. Analysis by Year of Establishment
5.2.2. Analysis by Company Size
5.2.3. Analysis by Location of Headquarters
5.2.4. Analysis by Type of Product(s) Manufactured
5.2.5. Analysis by Location of Vector Manufacturing Facilities
5.2.6. Analysis by Type of Manufacturer and Company Size
5.2.7. Analysis by Scale of Operation
5.2.8. Analysis by Location of Headquarters and Scale of Operation
5.2.9. Analysis by Application Area(s)

6. AAV TECHNOLOGY: MARKET LANDSCAPE
6.1. Chapter Overview
6.2. AAV Technology
6.2.1. Analysis by Type of Technology
6.2.2. Analysis by Scale of Operation
6.2.3. Analysis by Application Area(s)
6.2.4. Analysis by Therapeutic Area
6.3. AAV Technology: List of Developers
6.3.1. Analysis by Year of Establishment
6.3.2. Analysis by Company Size
6.3.3. Analysis by Location of Headquarters
6.3.4. Analysis by Company Size and Location of Headquarters
6.3.5. AAV Technology: Leading Developers

7. DRUG PROFILES
7.1. Chapter Overview
7.2. Marketed AAV Based Gene Therapies
7.2.1. LUXTURNA? (Spark Therapeutics)
7.2.1.1. Company Overview
7.2.1.2. Development Timeline
7.2.1.3. Mechanism of Action
7.2.1.4. Target Indication(s)
7.2.1.5. Current Status of Development
7.2.1.6. Manufacturing, Dosage and Sales
7.2.2. ZOLGENSMA? (Novartis)
7.2.2.1. Company Overview
7.2.2.2. Development Timeline
7.2.2.3. Mechanism of Action
7.2.2.4. Target Indication(s)
7.2.2.5. Current Status of Development
7.2.2.6. Manufacturing, Dosage and Sales
7.3.1. HEMGENIX? (CSL Behring / UniQure)
7.3.1.1. Company Overview
7.3.1.2. Development Timeline
7.3.1.3. Mechanism of Action
7.3.1.4. Target Indication(s)
7.3.1.5. Current Status of Development
7.3.1.6. Manufacturing, Dosage and Sales
7.4.1. ROCTAVIAN? (BioMarin Pharmaceuticals)
7.4.1.1. Company Overview
7.4.1.2. Development Timeline
7.4.1.3. Mechanism of Action
7.4.1.4. Target Indication(s)
7.4.1.5. Current Status of Development
7.4.1.6. Manufacturing, Dosage and Sales
7.5.1. ELEVIDYS? (Sarepta Therapeutics)
7.5.1.1. Company Overview
7.5.1.2. Development Timeline
7.5.1.3. Mechanism of Action
7.5.1.4. Target Indication(s)
7.5.1.5. Current Status of Development
7.5.1.6. Manufacturing, Dosage and Sales
7.6.1. UPSTAZA? (PTC Therapeutics)
7.6.1.1. Company Overview
7.6.1.2. Development Timeline
7.6.1.3. Mechanism of Action
7.6.1.4. Target Indication(s)
7.6.1.5. Current Status of Development
7.6.1.6. Manufacturing, Dosage and Sales
7.6. Late Stage (Phase II/III and Above) Gene Therapies
7.6.1. AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.6.2. LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.6.3. NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.6.4. RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.6.5. OAV-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.6.6. PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.6.7. PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.6.8. PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.6.9. SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results

8. COMPANY PROFILES
8.1. Chapter Overview
8.2. Abeona Therapeutics
8.2.1. Company Overview
8.2.2. Financial Information
8.2.3. AAV Manufacturing Related Capabilities
8.2.4. Recent Developments and Future Outlook
8.3 Aldevron (Acquired by Danaher)
8.3.1. Company Overview
8.3.2. Financial Information
8.3.3. AAV Manufacturing Related Capabilities
8.3.4. Recent Developments and Future Outlook
8.4 Oxford BioMedica
8.4.1. Company Overview
8.4.2. Financial Information
8.4.3. AAV Manufacturing Related Capabilities
8.4.4. Recent Developments and Future Outlook
8.5 Sanofi (CEPiA, Sanofi Pasteur, Genzyme)
8.5.1. Company Overview
8.5.2. Financial Information
8.5.3. AAV Manufacturing Related Capabilities
8.5.4. Recent Developments and Future Outlook
8.6. WuXi AppTec
8.6.1. Company Overview
8.6.2. Financial Information
8.6.3. AAV Manufacturing Related Capabilities
8.6.4. Recent Developments and Future Outlook
8.7. YPOSKESI
8.7.1. Company Overview
8.7.2. AAV Manufacturing Related Capabilities
8.7.3. Recent Developments and Future Outlook
9. COMPANY COMPETITIVENESS ANALYSIS
9.1. Chapter Overview
9.2. Methodology and Key Parameters
9.3. AAV Manufacturing Companies: In-house Manufacturers
9.3.1. Players based in North America
9.3.2. Players based in Europe
9.4. AAV Manufacturing Companies: Contract Manufacturing Organizations
9.4.1. Players based in North America
9.4.2. Players based in Europe
9.4.3. Players based in Asia-Pacific
9.5. AAV Manufacturing Companies: Both In-House and Contract Manufacturing Organizations
9.5.1. Players based in North America
9.5.2. Players based in Europe
9.5.3. Players based in Asia-Pacific
10. TECHNOLOGY COMPETITIVENESS ANALYSIS
10.1. Chapter Overview
10.2. Methodology and Key Parameters
10.3. AAV Technology Platforms
10.3.1. AAV Technology Platforms Developed by Companies based in North America
10.3.2. AAV Technology Platforms Developed by Companies based in Europe and Asia-Pacific
11. CLINICAL TRIAL ANALYSIS
11.1. Chapter Overview
11.2. Scope and Methodology
11.3. AAV Based Gene Therapy: Clinical Trial Analysis
11.3.1. Analysis by Trial Registration Year
11.3.2. Analysis by Trial Registration Year and Enrolled Patient Population
11.3.3. Analysis by Trial Phase and Enrolled Patient Population
11.3.4. Analysis by Trial Status
11.3.5. Analysis by Study Design
11.3.6. Analysis by Therapeutic Area
11.3.7. Analysis by Target Disease Indication
11.3.8. Analysis by Type of Sponsor / Collaborator
11.3.9. Leading Industry Players: Analysis by Number of Registered Trials
11.3.10. Regional Analysis
11.3.10.1. Analysis by Number of Registered Trials
11.3.10.2. Analysis by Enrolled Patient Population
12. PARTNERSHIPS AND COLLABORATIONS
12.1. Chapter Overview
12.2. Partnership Models
12.3. AAV Based Gene Therapy: List of Partnerships and Collaborations
12.3.1. Analysis by Year of Partnership
12.3.2. Analysis by Type of Partnership
12.3.3. Analysis by Year of Partnership and Type of Partner
12.3.4. Analysis by Type of Partnership and Type of Partner
12.3.5. Analysis by Therapeutic Area
12.3.6. Most Active Players: Analysis by Number of Partnerships
12.3.7. Regional Analysis
12.3.7.1. Intercontinental and Intracontinental Agreements
12.4. AAV Manufacturing Companies: List of Partnerships and Collaborations
12.4.1. Analysis by Year of Partnership
12.4.2. Analysis by Type of Partnership
12.4.3. Analysis by Year of Partnership and Type of Partner
12.4.4. Analysis by Type of Partnership and Type of Partner
12.4.5. Analysis by Therapeutic Area
12.4.6. Most Active Players: Analysis by Number of Partnerships
12.4.7. Regional Analysis
12.4.7.1. Intercontinental and Intracontinental Agreements

13. STRATEGIC PARTNER ANALYSIS
13.1. Chapter Overview
13.2. AAV Manufacturing Companies: Potential Strategic Partners
13.2.1. Methodology and Key Parameters
13.2.2. AAV Manufacturing Companies: Strategic Partner Analysis
13.2.2.1. Most Likely Partners
13.2.2.2. Likely Partners
13.2.2.3. Less Likely Partners
13.2.2.4. Least Likely Partners

14. PATENT ANALYSIS
14.1. Chapter Overview
14.2. Scope and Methodology
14.3. Adeno-Associated Viral Vectors: Patent Analysis
14.3.1. Analysis by Publication Year
14.3.2. Analysis by Annual Patent Application(s)
14.3.3. Analysis by Annual Granted Patents
14.3.4. Analysis by Geography
14.3.5. Analysis by CPC Symbols
14.3.6. Analysis by Type of Applicant
14.3.7. Leading Industry Players: Analysis by Number of Patents
14.4. Adeno Associated Viral Vector Based Market: Patent Benchmarking
14.4.1. Analysis by Patent Characteristics
14.5. Adeno-Associated Viral Vectors: Patent Valuation
14.6. Leading Patents: Analysis by Number of Citations

15. START-UP HEALTH INDEXING
15.1. Chapter Overview
15.2. Start-ups Developing Adeno-Associated Viral Vectors
15.2.1. Analysis by Location of Headquarters
15.3. Benchmarking of Start-ups
15.3.1. Analysis by Pipeline Strength
15.3.2. Analysis by Pipeline Maturity
15.3.3. Analysis by Indication Diversity
15.3.4. Analysis by Number of Patents
15.3.5. Analysis by Partnership Activity
15.3.6. Start-ups Health Indexing: Roots Analysis Perspective

16. OUTSOURCING: GO / NO-GO FRAMEWORK
16.1. Chapter Overview
16.2. Outsourcing: Go / No-Go Framework
16.3. AAV Based Gene Therapy Developers Outsourcing: Go / No-Go Framework
16.3.1. Key Parameters and Assumptions
16.3.2. Methodology
16.3.3. Results and Interpretations
16.3.3.1. Small Companies
16.3.3.2. Mid-sized Companies
16.3.3.3. Large Companies

17. MARKET SIZING AND OPPORTUNITY ANALYSIS
17.1. Chapter Overview
17.2. Methodology and Key Assumptions
17.3. AAV Based Gene Therapy Market, 2022-2035
17.3.1. AAV Based Gene Therapy Market: Distribution by Therapeutic Area
17.3.2. AAV Based Gene Therapy Market: Distribution by Type of Therapy
17.3.3. AAV Based Gene Therapy Market: Distribution by Type of Gene Delivery Method Used
17.3.4. AAV Based Gene Therapy Market: Distribution by Therapeutic Area
17.3.5. AAV Based Gene Therapy Market: Distribution by Route of Administration
17.3.6. AAV Based Gene Therapy Market: Distribution by Therapeutic Area
17.3.7. AAV Based Gene Therapy Market: Distribution by Geography
17.3.8. AAV Based Gene Therapy Market, 2022-2035: Individual Product Sales Forecast
17.3.8.1. LUXTURNA
17.3.8.1.1. Target Patient Population
17.3.8.1.2. Sales Forecast
17.3.8.1.3. Net Present Value
17.3.8.1.4. Value Creation Analysis
17.3.8.2. ZOLGENSMA
17.3.8.2.1. Target Patient Population
17.3.8.2.2. Sales Forecast
17.3.8.2.3. Net Present Value
17.3.8.2.4. Value Creation Analysis
17.3.8.3. HEMGENIX
17.3.8.3.1. Target Patient Population
17.3.8.3.2. Sales Forecast
17.3.8.3.3. Net Present Value
17.3.8.3.4. Value Creation Analysis
17.3.8.4. ROCTAVIAN
17.3.8.4.1. Target Patient Population
17.3.8.4.2. Sales Forecast
17.3.8.4.3. Net Present Value
17.3.8.4.4. Value Creation Analysis
17.3.8.5. UPSTAZA
17.3.8.5.1. Target Patient Population
17.3.8.5.2. Sales Forecast
17.3.8.5.3. Net Present Value
17.3.8.5.4. Value Creation Analysis
17.3.8.6. ELEVIDYS
17.3.8.6.1. Target Patient Population
17.3.8.6.2. Sales Forecast
17.3.8.6.3. Net Present Value
17.3.8.6.4. Value Creation Analysis
17.3.8.7. AGTC 501
17.3.8.7.1. Target Patient Population
17.3.8.7.2. Sales Forecast
17.3.8.7.3. Net Present Value
17.3.8.7.4. Value Creation Analysis
17.3.8.8. LYS-SAF302
17.3.8.8.1. Target Patient Population
17.3.8.8.2. Sales Forecast
17.3.8.8.3. Net Present Value
17.3.8.8.4. Value Creation Analysis
17.3.8.9. NFS-01
17.3.8.9.1. Target Patient Population
17.3.8.9.2. Sales Forecast
17.3.8.9.3. Net Present Value
17.3.8.9.4. Value Creation Analysis
17.3.8.10. OAV-101
17.3.8.10.1. Target Patient Population
17.3.8.10.2. Sales Forecast
17.3.8.10.3. Net Present Value
17.3.8.10.4. Value Creation Analysis
17.3.8.11. PF-06838435
17.3.8.11.1. Target Patient Population
17.3.8.11.2. Sales Forecast
17.3.8.11.3. Net Present Value
17.3.8.11.4. Value Creation Analysis
17.3.8.12. PF-06939926
17.3.8.12.1. Target Patient Population
17.3.8.12.2. Sales Forecast
17.3.8.12.3. Net Present Value
17.3.8.12.4. Value Creation Analysis
17.3.8.13. PF-07055480
17.3.8.13.1. Target Patient Population
17.3.8.13.2. Sales Forecast
17.3.8.13.3. Net Present Value
17.3.8.13.4. Value Creation Analysis
17.3.8.14. RGX-314
17.3.8.14.1. Target Patient Population
17.3.8.14.2. Sales Forecast
17.3.8.14.3. Net Present Value
17.3.8.14.4. Value Creation Analysis
17.3.8.15. SPK-8011
17.3.8.15.1. Target Patient Population
17.3.8.15.2. Sales Forecast
17.3.8.15.3. Net Present Value
17.3.8.15.4. Value Creation Analysis
17.4. AAV Manufacturing Market, 2022-2035
17.4.1. AAV Manufacturing Market, 2022-2035: Distribution by Phase of Development
17.4.2. AAV Manufacturing Market, 2022-2035: Distribution by Therapeutic Area
17.4.3. AAV Manufacturing Market, 2022-2035: Distribution by Application Area
17.4.4. AAV Manufacturing Market, 2022-2035: Distribution by Key Geographical Regions
17.5 Current and Future Market Opportunity for Commercial Products
17.5.1. AAV Manufacturing Market for Commercial Products, 2022-2035: Distribution by Therapeutic Area
17.5.2. AAV Manufacturing Market for Commercial Products, 2022-2035: Distribution by Application Area
17.5.3. AAV Manufacturing Market for Commercial Products, 2022-2035: Distribution by Key Geographical Regions
17.6. Current and Future Market Opportunity for Clinical Candidates
17.6.1. AAV Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Phase of Development
17.6.2. AAV Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Application Area
17.6.3. AAV Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Key Geographical Regions
17.7. Current and Future Market Opportunity for Preclinical Candidates
17.7.1. AAV Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Type of Animal Model Used
17.7.2. AAV Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Application Area
17.7.3. AAV Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Key Geographical Regions
18. CONCLUDING REMARKS
19. EXECUTIVE INSIGHTS
19.1. Chapter Overview
19.2. Boston College
19.2.1. Organization Snapshot
19.2.2. Interview Transcript: Abhishek Chatterjee, Professor
19.3. University of Florida
19.3.1. Organization Snapshot
19.3.2. Interview Transcript: Arun Srivastava, Professor of Genetics and Chief of Division of Cellular & Molecular Therapy
19.4. Andelyn Biosciences
19.4.1. Organization Snapshot
19.4.2. Interview Transcript: Adam Lauber, Chief Financial Officer
20. APPENDIX 1: TABULATED DATA
21. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS