LAS VEGAS, Jan. 25, 2021 /PRNewswire/ -- The AAV Vector-Based Gene Therapy market size is anticipated to shoot up exponentially attributing to an increase in the approval of a growing number of gene therapies and readily adoption on approval, ability to treat a broad array of conditions, increasing prevalence of diseases, convenient one-time dosing approach and curative treatment options.

DelveInsight's Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Marketreport offers a clear picture of the market position of the AAV vectors in the Gene therapy, emerging pipeline therapies, AAV vector-based Gene Therapy market share occupied by individual diseases, current and forecasted market share in the 7MM (the US, EU5 (the UK, Germany, France, Italy and Spain) and Japan) for the study period 2017-30.

The Report highlights the drivers and constraints shaping the present AAV Vector-Based Gene Therapy marketalong with the unmet medical needs that offer opportunities to the key players to explore the underlying potential of the market.

Key Highlights of AAV Vector-Based Gene Therapy Market:

Know more about the report highlights @ Adeno-associated virus (AAV) Vector-Based Gene Therapy Market Landscape

Adeno-Associated Virus (AAV)

Adeno-associated virus hails from the genus Dependoparvovirus, which in turn belongs to the family Parvoviridae. They are small viruses (25-nm) with a genome of single-stranded DNA (~4.7kb) that can either be the plus (sense) or minus (anti-sense) strand. AAV is replication-defective and depends on a helper virus for effective and productive replication in mammalian cells. However, for choosing AAV as a gene delivery vector depends on:

Adeno-associated virus (AAV) Vector-Based Gene Therapy

With advances in bioengineering and genetics, the horizon of the medical approaches giving rise to novel treatment options, such as Gene therapy has also widened. Gene therapyhas emerged out as a promising treatment approach for a number of inherited disorders, certain types of cancer, and certain viral infections. The delivery of gene therapy involves "vectors" which can be either viral or non-viral vectors. Out of the several viral vectors, Adeno-associated virus (AAV) vectors are currently among the most frequently used, safest and effective viral vectors for gene therapy delivery. AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases.

Key Indications:

Haematology

Ophthalmology

Lysosomal Storage Disorders

Neurology

Neurological Disorders

Musculoskeletal

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Epidemiology Segmentation

In the year 2020, the total prevalent cases of selected indications in which AAV Gene Therapies were administered were estimated to be 2,863,103 in the 7MM. DelveInsight's Adeno-Associated Virus Vector-Based Gene Therapy MarketReport provides historical as well as forecasted epidemiological analysis for the study period 2017-30 for the 7MM segmented into:

Adeno-Associated Virus Vector-Based Gene Therapy Market

At the moment, the AAV Vector-Based Gene Therapy Markethas only two FDA-approved AAV vector-based gene therapies approved.

Spark Therapeutics' Luxturna is approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. It is the first FDA-approved gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease and the first AAV vector gene therapy approved in the United States.

Another therapy occupying the market share is Novartis/AveXis's Zolgensma, an AAV-delivered gene therapy indicated for the treatment of paediatric patients <2 years of age with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Currently, it is being investigated in the global Phase III STR1VE clinical program (consisting of STR1VE-US, STR1VE-EU, and STR1VE-AP) to evaluate the intravenous (IV) formulation of AVXS-101 in patients who have SMA Type 1, and the multinational Phase III SPR1NT trial in presymptomatic patients who have a genetic diagnosis of SMA with two or three copies of the SMN2 gene.

However, before Luxturna and Zolgensma managed to steal the spotlight in the AAV Vector-Based Gene Therapy Market,there was another candidate, Glybera, which got the nod from the EMA to treat an ultra-rare, hereditary lipoprotein lipase deficiency(LPLD); and became the first Gene therapy to get launched. However, the decision of the company to not renew its market authorization after it expired in October 2017 stresses on the cost of the maintenance and development of the gene therapies even if they prove to be outright cures.

DelveInsight estimates that the Adeno-Associated Virus Vector-Based Gene Therapy Market,is anticipated to pick up momentum as companies across the globe are thoroughly working toward the development of new AAV-gene therapies to treat a spectrum of diseases. Key pharmaceutical and biotech companies such as Biomarin Pharmaceutical, Roche (Spark Therapeutics), Sangamo, Pfizer among several others are involved in exploring the AAV vector-based gene therapy for a wide range of indications such as Hemophilia A and B, MPS, and others.

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Key Pipeline Therapies:

There is not a shred of doubt that clinical successes in AAV-mediated gene replacement have helped Adeno-Associated Viruses to get recognized as an ideal therapeutic vector for delivery of gene therapies. With two AAV vector-based gene therapies having won regulatory approval,their popularity as the predominant vectors that deliver genes of interest to target tissues with improved specificity, efficiency, and safety in the Gene therapy market further fuelled. However, the production and formulation of AAV products require specified conditions so as to ensure good stability and yield, and even after caution, some of its processing methods (filtration or lyophilization) may lead to aggregation or loss of AAV titer. Further, storing the AAV products can also prove to be quite challenging.

Conclusively, Gene therapy has proved to be an ingenious tool in the healthcare sector and with dramatic advancements being made in the domain, its potential is unfolding at a rapid pace. Thus, it is not wrong to say that Gene therapy is set to emerge as a novel therapeutic option.

Scope of the report:

Table of Content

1

Key Insights

2

Executive Summary of Adeno-Associated Virus Vectors in Gene Therapy

3

Competitive Intelligence Analysis for AAV Vector-Based Gene Therapy Market

4

AAV Vector-Based Gene Therapy Market SWOT Analysis

5

Adeno-Associated Virus Vector-Based Gene Therapy Market Overview at a Glance

6

Adeno-Associated Virus Vector-Based Gene Therapy: Disease Background and Overview

7

Patient Journey

8

Adeno-Associated Virus Vectors in Gene Therapy Epidemiology and Patient Population

9

AAV Vector-Based Gene Therapy Market: Treatment Algorithm, Current Treatment, and Medical Practices

10

AAV Vector-Based Gene Therapy Market Unmet Needs

11

Key Endpoints of Adeno-Associated Virus Vectors in Gene Therapy Treatment

12

Marketed Products in AAV Vector-Based Gene Therapy Market

13

Emerging Adeno-Associated Virus Vector-Based Gene Therapies

14

Adeno-Associated Virus Vectors in Gene Therapy: Seven Major Market Analysis

15

Attribute analysis

16

Adeno-Associated Virus Vector-Based Gene Therapy Market Outlook: 7MM

17

Access and Reimbursement Overview of Adeno-Associated Virus Vector-Based Gene Therapy Market

18

KOL Views

19

Adeno-Associated Virus Vector-Based Gene Therapy Market Drivers

20

Adeno-Associated Virus Vector-Based Gene Therapy Market Barriers

21

Appendix

22

DelveInsight Capabilities

23

Disclaimer

24

About DelveInsight

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Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Expected to Grow at a Significant CAGR of 43.4% During the Study Period 2019-30 -...

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