Table of Content

1. Key Insights

2. Key Highlights from Report

3. Executive Summary of Adeno-Associated Virus (AAV) Vector Based Gene Therapy

4. AAV Vector Based Gene Therapy: Market Share (%) Distribution Overview at a Glance: By Country

5. Epidemiology and Market Methodology

6. Adeno-Associated Virus (AAV) Vector Based Gene Therapy: Background and Overview
6.1. Introduction
6.2. Viral Vectors
6.3. Adeno-Associated Virus (AAV): Biology
6.4. Adeno-associated Virus (AAV): Vector
6.4.1. rAAV transduction pathway
6.5. rAAV gene therapy strategies
6.5.1. Gene replacement
6.5.2. Gene silencing
6.5.3. Gene addition
6.5.4. Gene editing
6.6. AAV serotypes and Tissue tropism
6.7. Pros and Cons of AAV Gene Therapy
6.8. AAV-vector gene therapy in Clinical Trials
6.8.1. Eye diseases
6.8.2. Hemophilia
6.8.3. Neurological diseases
6.8.4. Muscular diseases
6.8.5. Heart diseases
6.8.6. Other disorders
6.9. Considerations for successful AAV-mediated gene therapies

7. Epidemiology and Patient Population
7.1. Epidemiology Key Findings
7.2. Assumptions and Rationale: 7MM

8. Epidemiology Scenario: 7MM
8.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in the 7MM
8.2. Total Indication wise eligible cases in the 7MM
8.3. Indication wise Treated Cases of AAV Gene Therapies in the 7MM

9. The United States Epidemiology
9.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in the United States
9.2. Total Indication wise eligible cases in the United States
9.3. Indication wise Treated Cases of AAV Gene Therapies in the United States

10. EU-5 Epidemiology
10.1. Germany
10.1.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in Germany
10.1.2. Total Indication wise eligible cases in Germany
10.1.3. Indication wise Treated Cases of AAV Gene Therapies in Germany
10.2. France
10.2.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in France
10.2.2. Total Indication wise eligible cases in France
10.2.3. Indication wise Treated Cases of AAV Gene Therapies in France
10.3. Italy
10.3.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in Italy
10.3.2. Total Indication wise eligible cases in Italy
10.3.3. Indication wise Treated Cases of AAV Gene Therapies in Italy
10.4. Spain
10.4.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in Spain
10.4.2. Total Indication wise eligible cases in Spain
10.4.3. Indication wise Treated Cases of AAV Gene Therapies in Spain
10.5. The United Kingdom
10.5.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in the United Kingdom
10.5.2. Total Indication wise eligible cases in the United Kingdom
10.5.3. Indication wise Treated Cases of AAV Gene Therapies in the United Kingdom

11. Japan Epidemiology
11.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in Japan
11.2. Total Indication wise eligible cases in Japan
11.3. Indication wise Treated Cases of AAV Gene Therapies in Japan

12. Appendix
12.1. Bibliography
12.2. Report Methodology

13. DelveInsight Capabilities

14. Disclaimer

15. About DelveInsight



List of Figures


Figure 1 AAV Vector
Figure 2 Gene Therapy – Therapeutic Gene Delivery via Viral Vector
Figure 3 Examples of viral vectors applied for gene therapy
Figure 4 Timeline of AAV Gene Therapies
Figure 5 AAV-2 Genome Map
Figure 6 AAV vector in gene therapy
Figure 7 Gene therapy strategies
Figure 8 Tissue tropisms by AAV serotypes
Figure 9 Spinal muscular atrophy gene therapy
Figure 10 Neurodegenerative diseases
Figure 11 Total Prevalent Cases of selected indications for AAV Gene Therapies in the 7MM (2017–2030)
Figure 12 Total Indication wise eligible cases in the 7MM (2017–2030)
Figure 13 Indication wise Treated Cases of AAV Gene Therapies in the 7MM (2017–2030)
Figure 14 Total Prevalent Cases of selected indications for AAV Gene Therapies in the United States (2017–2030)
Figure 15 Total Indication wise eligible cases in the United States (2017–2030)
Figure 16 Indication wise Treated Cases of AAV Gene Therapies in the United States (2017–2030)
Figure 17 Total Prevalent Cases of selected indications for AAV Gene Therapies in Germany (2017–2030)
Figure 18 Total Indication wise eligible cases in Germany (2017–2030)
Figure 19 Indication wise Treated Cases of AAV Gene Therapies in Germany (2017–2030)
Figure 20 Total Prevalent Cases of selected indications for AAV Gene Therapies in France (2017–2030)
Figure 21 Total Indication wise eligible cases in France (2017–2030)
Figure 22 Indication wise Treated Cases of AAV Gene Therapies in France (2017–2030)
Figure 23 Total Prevalent Cases of selected indications for AAV Gene Therapies in Italy (2017–2030)
Figure 24 Total Indication wise eligible cases in Italy (2017–2030)
Figure 25 Indication wise Treated Cases of AAV Gene Therapies in Italy (2017–2030)
Figure 26 Total Prevalent Cases of selected indications for AAV Gene Therapies in Spain (2017–2030)
Figure 27 Total Indication wise eligible cases in Spain (2017–2030)
Figure 28 Indication wise Treated Cases of AAV Gene Therapies in Spain (2017–2030)
Figure 29 Total Prevalent Cases of selected indications for AAV Gene Therapies in the United Kingdom (2017–2030)
Figure 30 Total Indication wise eligible cases in the United Kingdom (2017–2030)
Figure 31 Indication wise Treated Cases of AAV Gene Therapies in the United Kingdom (2017–2030)
Figure 32 Total Prevalent Cases of selected indications for AAV Gene Therapies in Japan (2017–2030)
Figure 33 Total Indication wise eligible cases in Japan (2017–2030)
Figure 34 Indication wise Treated Cases of AAV Gene Therapies in Japan (2017–2030)

 

List of Tables


Table 1 Summary of AAV Vectors in Gene Therapy, , Epidemiology, and Key Events (2019–2030)
Table 2 List of sources Used for Epidemiology Evaluation for shortlisted Indication
Table 3 Total Prevalent Cases of selected indications for AAV Gene Therapies in the 7MM (2017–2030)
Table 4 Total Indication wise eligible cases in the 7MM (2017–2030)
Table 5 Total Indication wise Treated Cases of AAV Gene Therapies in the 7MM (2017–2030)
Table 6 Total Prevalent Cases of selected indications for AAV Gene Therapies in the United States (2017–2030)
Table 7 Total Indication wise eligible cases in the United States (2017–2030)
Table 8 Total Indication wise Treated Cases of AAV Gene Therapies in the United States (2017–2030)
Table 9 Total Prevalent Cases of selected indications for AAV Gene Therapies in Germany (2017–2030)
Table 10 Total Indication wise eligible cases in Germany (2017–2030)
Table 11 Total Indication wise Treated Cases of AAV Gene Therapies in Germany (2017–2030)
Table 12 Total Prevalent Cases of selected indications for AAV Gene Therapies in France (2017–2030)
Table 13 Total Indication wise eligible cases in France (2017–2030)
Table 14 Total Indication wise Treated Cases of AAV Gene Therapies in France (2017–2030)
Table 15 Total Prevalent Cases of selected indications for AAV Gene Therapies in Italy (2017–2030)
Table 16 Total Indication wise eligible cases in Italy (2017–2030)
Table 17 Total Indication wise Treated Cases of AAV Gene Therapies in Italy (2017–2030)
Table 18 Total Prevalent Cases of selected indications for AAV Gene Therapies in Spain (2017–2030)
Table 19 Total Indication wise eligible cases in Spain (2017–2030)
Table 20 Total Indication wise Treated Cases of AAV Gene Therapies in Spain (2017–2030)
Table 21 Total Prevalent Cases of selected indications for AAV Gene Therapies in the United Kingdom (2017–2030)
Table 22 Total Indication wise eligible cases in the United Kingdom (2017–2030)
Table 23 Total Indication wise Treated Cases of AAV Gene Therapies in the United Kingdom (2017–2030)
Table 24 Total Prevalent Cases of selected indications for AAV Gene Therapies in Japan (2017–2030)
Table 25 Total Indication wise eligible cases in Japan (2017–2030)
Table 26 Total Indication wise Treated Cases of AAV Gene Therapies in Japan (2017–2030)