Adeno-Associated Virus (AAV) Vectors in Gene Therapy Epidemiology Insights Report 2021-2030 –

The "Adeno-Associated Virus (AAV) Vectors in Gene Therapy – Epidemiology Insight – 2030" report has been added to’s offering.
This ‘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.
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AAV-based gene therapy, Glybera, was approved by the European Medicines Agency (EMA) in 2012 but later in 2017, it was withdrawn from the market mainly due to commercial failure.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
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 report also provides the diagnosed patient pool and their trends along with assumptions undertaken.
Key Topics Covered:
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
10. EU-5 Epidemiology
10.1. Germany
10.2. France
10.3. Italy
10.4. Spain
10.5. 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
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PUB: 01/27/2021 12:56 PM/DISC: 01/27/2021 12:55 PM