Adeno Associated Virus Vectors In Gene Therapy Epidemiology Forecast

DelveInsight’s ‘Adeno-Associated Virus (AAV) Vector Based Gene Therapy- Epidemiology Forecast to 2030’ report delivers an in-depth understanding of the AAV vector based gene therapy across the various therapeutic areas are several selected diseases, historical and forecasted AAV Vector Based Gene Therapy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.


Geographies Covered

• The United States

• EU5 (Germany, France, Italy, Spain, and the United Kingdom)

• Japan


Study Period: 2017–2030


Adeno-Associated Virus (AAV) Vector Based Gene Therapy Understanding

Gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections). Gene therapy involves “vectors” which can be either viral or non-viral vectors. Out of the several viral vectors that have been used to date for delivering the genes of interest, the Adeno-associated viral (AAV) vector appears to be the safest and effective vehicle and can maintain long-term gene and protein expression following a single injection of the vector. There are currently two classes of recombinant AAVs (rAAVs) in use: single-stranded AAV (ssAAV) and self-complementary AAV (scAAV). rAAV gene therapy strategies include Gene replacement, Gene silencing, Gene addition and Gene editing. There are 12 naturally occurring serotypes (AAV serotype 1 [AAV-1] to AAV-12)and more than 100 variants of AAV, each of which differs in its amino acid sequence, particularly within the hypervariable regions of the capsid proteins, and, thus, also differ slightly in their gene delivery properties. AAV2 is the most extensively studied serotype among all. The tissue tropism of the different AAV serotypes is determined by the different cell surface receptors used for the attachment to the target cell. According to certain comparative studies between AAV serotypes, when it comes to targeting tissues, AAV3 and AAV4 are the slowest, and among all the serotypes, AAV2, 3, 4, and 5 have the lowest transduction efficiency.


The first AAV-based gene therapy, Glybera, was approved by the European Medicines Agency (EMA) in 2012 but later in 2017, it was withdrawn from the market mainly due to commercial failure. Only two AAV-based gene therapies are currently FDA-approved. Luxturna was approved in 2017 for a rare inherited retinal dystrophy, and Zolgensma was approved in 2019 for spinal muscular atrophy. Keeping in mind, the numerous advantages of AAV-vectors, currently, these vectors are being tested to cure diseases which were earlier thought to be nearly impossible to treat. Be it any Ocular disorder, blood disorder, metabolic disorder or muscular disorder expectations are high from AAV-vector gene therapy and thus, clinical trials for each mentioned disorders are positively going on.


Epidemiology Perspective by DelveInsight

The Adeno-Associated Virus (AAV) vector based gene therapy epidemiology division provides the insights about historical and current indication wise eligible patient pool and forecasted trend for each seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.


Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted AAV Gene Therapies epidemiology [segmented as Total cases of AAV Gene Therapy Targeted Indications, Total diagnosed indication-specific cases, Number of Patients Eligible for AAV Gene Therapy and Total treated cases] scenario of AAV Gene Therapy in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.


Country Wise- Adeno-Associated Virus (AAV) vector based gene therapy Epidemiology

  • In the year 2017, the total prevalent cases of selected indications for AAV Gene Therapies were 2,718,559 cases in the 7MM which are expected to grow during the study period, i.e., 2017–2030.
  • In the United States, the total number of prevalent cases of AAV vector based gene therapy was 1,333,147 cases in the year 2017 which are expected to grow during the study period, i.e., 2017–2030.
  • In EU-5 countries the highest number of prevalent cases of AAV vector based gene therapy were in Germany, i.e., 428,198 in the year 2017 which are expected to grow during the study period, i.e., 2017–2030.
  • In Japan, the total number of prevalent cases of AAV vector based gene therapy was 233,892 cases in the year 2017 which are expected to grow during the study period, i.e., 2017–2030.
  • Also, in the 7MM, the total treated cases of indication wise AAV Gene Therapies were 736,173 in the year 2017 which are expected to grow during the study period, i.e., 2017–2030.


Scope of the Report

  • The Adeno-Associated Virus (AAV) vector based gene therapy report covers a detailed overview of the development of AAV vectors and gene therapies based on them across the various therapeutic areas and several selected indications, explaining its various serotypes, benefits, and challenges for future.
  • The AAV vector based gene therapy Report and Model provide an overview of the risk factors and global trends of AAV vector based gene therapy in the seven major markets (7MM: United States, Germany, France, Italy, Spain, and the United Kingdom, and Japan)
  • The report provides insight about the historical and forecasted patient pool of AAV vector based gene therapy in seven major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
  • The report helps to recognize the growth opportunities in the 7MM with respect to the patient population.
  • The report assesses the selected disease risk and burden and highlights the unmet needs of AAV vector based gene therapy.
  • The report provides the segmentation of the AAV vector based gene therapy epidemiology by targeted Indications in the 7MM.
  • The report provides the segmentation of the AAV vector based gene therapy epidemiology by diagnosed indication-specific cases in the 7MM.
  • The report provides the segmentation of the AAV vector based gene therapy epidemiology by number of Patients Eligible for AAV Gene Therapy in the 7MM.
  • The report provides the segmentation of the AAV vector based gene therapy epidemiology by treated cases of AAV vector based gene therapy in the 7MM.


Report Highlights

  • 11-Year Forecast
  • 7MM Coverage
  • Total Prevalent Cases of AAV gene therapy targeted indications.
  • Total diagnosed indication-specific cases of AAV vector based gene therapy.
  • Number of patients eligible for AAV vector based gene therapy
  • Total Treated cases of AAV vector based gene therapy


KOL-Views

We interview, KOL’s and SME's opinion through primary research to fill the data gaps and validate our secondary research. The opinion helps to understand the total patient population and current treatment pattern. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the indications.


Key Questions Answered

  • What will be the growth opportunities in the 7MM with respect to the patient population pertaining to AAV vector based gene therapy?
  • What are the key findings pertaining to the AAV vector based gene therapy epidemiology across the 7MM and which country will have the highest number of prevalent cases of selected indications for AAV Gene Therapies the study period (2017–2030)?
  • What would be the total number of patients of AAV vector based gene therapy across the 7MM during the study period (2017–2030)?
  • Among the EU5 countries, which country will have the highest prevalent cases of selected indications for AAV Gene Therapies during the study period (2017–2030)?
  • At what CAGR the patient population is expected to grow in the 7MM during the study period (2017–2030)?
  • What are the various recent and upcoming events which are expected to improve the uptake of AAV Gene Therapies?


Reasons to buy

The AAV vector based gene therapy Epidemiology report will allow the user to -

  • Develop business strategies by understanding the trends shaping and driving the global AAV vector based gene therapy market
  • Quantify patient populations in the global AAV vector based gene therapy market to improve product design, pricing, and launch plans
  • Organize sales and marketing efforts by identifying the age groups and gender that present the best opportunities for AAV vector based gene therapy therapeutics in each of the markets covered
  • The AAV vector based gene therapy epidemiology report and model were written and developed by Masters and PhD level epidemiologists
  • The AAV vector based gene therapy Epidemiology Model developed by DelveInsight is easy to navigate, interactive with dashboards, and epidemiology based with transparent and consistent methodologies. Moreover, the model supports data presented in the report and showcases disease trends over 11-year forecast period using reputable sources


Key Assessments

  • Patient Segmentation
  • Selected Disease Risk and Burden
  • Factors driving growth in a specific disease patient population

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 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)

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)

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