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Adeno-Associated Virus (AAV) Vectors in Gene Therapy - Epidemiology Insight - 2030

Adeno-Associated Virus (AAV) Vectors in Gene Therapy - Epidemiology Insight - 2030

  • January 2021
  • 202 pages
  • ID: 6010479
  • Format: PDF
  • Delve Insight

Summary

Table of Contents

‘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AAV-based gene therapy, Glybera, was approved by the European Medicines Agency (EMA) in 2012 but later in 2017, it was withdrawn from the market mainly due to commercial failure.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 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 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

Geographies Covered 
• The United States 
• EU5 (Germany, France, Italy, Spain, and the United Kingdom) 
• Japan 
Study Period: 2017–2030

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