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Global Adeno-Associated Virus Vector-Based Gene Therapy Market Share, Size, Trends: By Therapeutic Agents: Corticosteroid Drugs, Immunosuppressive Drugs, ACE Inhibitors and ARBs, Others; By Indication Type: Hematological Disorders, Others; By Treatment Channel; Regional Analysis; Market Dynamics; Supplier Landscape; 2023-2031

Global Adeno-Associated Virus Vector-Based Gene Therapy Market

The adeno-associated virus vector-based gene therapy market size was estimated to be worth USD 0.8 billion. Driven by the advancements in biotechnological and genetics sector, the market is expected to rise at a growth rate of 18.5% in the forecast period of 2023-2031, and by 2030, the market value is predicted to be worth USD 3.6 billion.

 

Adeno-Associated Virus: Insights, Viral Vectors, and Applications

Adeno-associated virus (AAV) is a group of viruses infecting humans and some other species. Adenovirus belongs to the family Parvoviridae. These viruses do not cause any disease but show a mild immune response.

 

The incorporation of adenovirus in viral vectors has supplied enormous therapeutic effects. These adeno-associated virus vectors provide gene therapies for several indications. AAV vectors are also used for the creation of human isogenic disease models.

 

The adeno-associated virus is used as a delivery system to transport the therapeutic genetic material into the living tissues. The AAV is considered to be the best candidate for viral vectors. This group of viruses neither cause any disease nor self-replicate in the body like other viruses.

 

Adeno-associated virus is the best choice for emerging gene therapies. Two FDA-approved treatments use adeno-associated viral vectors.

 

According to the adeno-associated virus vector-based gene therapy market research report, the market can be categorised into the following segments:

 

Market Breakup by Therapeutic Agents

  • Corticosteroid Drugs
  • Immunosuppressive Drugs
  • ACE Inhibitors and ARBs
  • Others

 

Market Breakup by Indication Type

  • Haematological Disorders
    • Haemophilia A
    • Haemophilia B
  • Ophthalmological Disorders
    • Chloridaemia
    • Retinitis Pigmentosa
  • Lysosomal Storage Disorders
    • Fabry Disease
    • Pompe Disease
    • Hunter Syndrome
  • Neurological Disorders
    • Parkinson's Disease
    • Batten Disease
  • Musculoskeletal Disorders
    • Duchenne Muscular Dystrophy (DMD)
    • Spinal Muscular Atrophy (SMA)

 

Market Breakup by Treatment Channel

  • Public
  • Private

 

Market Breakup by Region

  • North America
    • United States of America
    • Canada
  • Europe
    • United Kingdom
    • Germany
    • France
    • Italy
    • Others
  • Asia Pacific
    • China
    • Japan
    • India
    • ASEAN
    • Australia
    • Others
  • Latin America
    • Brazil
    • Argentina
    • Mexico
    • Others
  • Middle East and Africa
    • Saudi Arabia
    • United Arab Emirates
    • Nigeria
    • South Africa
    • Others

 

Market Scenario

Advancements in the Biotechnological and Genetics Sector

The advancements in the biotechnological and genetics sector have primarily driven the market growth. The novel technologies have discovered a new range of therapeutic products. These innovative approaches have widened gene therapies for a wide array of indications. This has led to the expansion of the adeno-associated virus vector-based gene therapy market.

 

Adeno-associated Virus Vectors: Most Effective Viral Vectors

Adeno-associated virus vectors are the safest and most effective viral vectors and are majorly used for emerging therapies. Due to their lucrative benefits, the market is predicted to witness significant growth in the forecast period.

 

Treats a Wide Array of Diseases

With extensive research, the incorporation of adeno-associated virus vectors in different therapeutic sectors is increasing. This application of AAV vectors in a wide range of diseases is expected to stimulate the growth of the market.

 

North America Accounts for the Largest Share

North America is anticipated to dominate all the regional markets. This is due to technological advancements, more significant expenditure in the healthcare sector, and an increasing number of key players. The rising gene therapy approvals also ease the development of the adeno-associated virus vector-based gene therapy market.

 

Therapeutic Landscape

The integration of adeno-associated viral vectors in gene therapy has paved the way for many novel treatments and medications. The AAV vector helps deliver the target drug to the target site, thus, aiding in managing the disease.

 

Glybera, the first therapy involving a ssAAV1 vector, was approved in 2012. European Medicine Agency approved this therapy for managing non-curable genetic disorders.

 

After a few years, the Food and Drug Administration approved the two novel AAV-derived therapies for managing uncurable disorders. Zolgensma and Luxturna are the two therapeutic products that have opened new therapeutic opportunities for a wide range of clinical indications. The vectors, namely ssAAV1 and ssAV9, are used for delivering the Zolgensma and Luxturna, respectively.
 

Recent advancements in gene modification tools and the joint application of AAV vectors have paved the way for novel therapeutic procedures. This will expand the product portfolio and attribute to the growth of the adeno-associated virus vector-based gene therapy market.

 

Clinical Trials Landscape

With extensive research and development, scientists and researchers have discovered some novel therapeutic interventions. Since the last decade, clinical trials have significantly increased due to innovations in genetic technologies.

 

The clinical testing for ocular gene treatments has tremendously increased in the last five years. The eye is considered the best organ due to its small tissue size, easy accessibility, and enclosed structure. The ongoing trials are in distinct phases of preclinical and clinical development.

 

AAV Vector Based Emerging Therapies and Drugs

BioMarin Pharmaceutics, a key player, is performing clinical trials for its emerging gene therapy Valoctocogene Roxaparvovec. In 2020, the company-initiated trials to evaluate the safety and efficacy of the treatment of Haemophilic patients.

 

The trials for the new therapy that delivers the human factor VIII gene through adeno-associated virus vectors are under Phase III. They can soon be brought to the market for patient use.

 

Freeline Therapeutics has discovered a novel gene therapy, FLT190. This therapy uses AAV8 to deliver a healthy copy of the GLA gene. The preclinical studies have shown the best safety and efficacy and thus seem promising for managing genetic disorders. Advanced clinical trials are needed for further evaluation.

 

The significant increase in the number of clinical trials has shifted the focus of the key players toward this sector. The approval of these therapies will increase the product portfolio, thus facilitating the development of the market.

 

Competitive Landscape

The report gives an in-depth analysis of the key players involved in the adeno-associated virus vector-based gene therapy market, sponsors manufacturing the therapies, and putting them through trials to get FDA approvals. The companies included in the market are as follows:

  • Arya Sciences Acquisition Corp.
  • Pfizer Inc.
  • Roche Holding AG
  • BioMarin Pharmaceutical Inc.
  • Bayer AG
  • Coave Therapeutics
  • MeiraGTx Limited
  • Neurocrine Biosciences, Inc.
  • Biogen, Inc.
  • Sangamo Therapeutics
  • Sarepta Therapeutics, Inc.

 

Key Highlights of the Report

REPORT FEATURES DETAILS
Base Year 2022
Historical Period 2016-2022
Forecast Period 2023-2031
Scope of the Report

Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:

  • Therapeutic Agents
  • Indication Type
  • Treatment Channel
  • Region
Breakup by Therapeutic Agents
  • Corticosteroid Drugs
  • Immunosuppressive Drugs
  • ACE Inhibitors and ARBs
  • Others
Breakup by Indication Type
  • Hematological Disorders
  • Ophthalmological Disorders
  • Lysosomal Storage Disorders
  • Neurological Disorders
  • Musculoskeletal Disorders
Breakup by Treatment Channel
  • Public
  • Private
Breakup by Region
  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East and Africa
Market Dynamics
  • SWOT
  • Porter's Five Forces
  • Key Indicators for Demand
  • Key Indicators for Price
Supplier Landscape
  • Market Structure
  • Company Profiles
    • Company Overview
    • Product Portfolio
    • Demographic Reach and Achievements
    • Certifications
Companies Covered
  • Arya Sciences Acquisition Corp.
  • Pfizer Inc.
  • Roche Holding AG
  • BioMarin Pharmaceutical Inc.
  • Bayer AG
  • Coave Therapeutics
  • MeiraGTx Limited
  • Neurocrine Biosciences, Inc.
  • Biogen, Inc.
  • Sangamo Therapeutics
  • Sarepta Therapeutics, Inc.

 

*At Expert Market Research, we strive to always give you current and accurate information. The numbers depicted in the description are indicative and may differ from the actual numbers in the final EMR report.

1    Preface
    1.1    Objectives of the Study
        1.1.1    Research Objectives
        1.1.2    Key Findings of the Report
    1.2    Limitations of the Study and Scope for Future Research
2    Research Methodology
3    Executive Summary
4    Global Adeno-Associated Virus Vector-Based Gene Therapy Overview 

    4.1    Guidelines and Stages
    4.2    Pathophysiology
    4.3    Screening and Diagnosis
    4.4    Treatment Pathway 
5    Patient Profile
    5.1    Patient Profile Overview 
    5.2    Patient Psychology and Emotional Impact Factors
    5.3    Risk Assessment and Treatment Success Rate
6    Global Adeno-Associated Virus Vector-Based Gene Therapy Market 
    6.1    Global Adeno-Associated Virus Vector-Based Gene Therapy Market Overview
    6.2    Global Adeno-Associated Virus Vector-Based Gene Therapy Market Analysis 
        6.2.1    Market Overview
            6.2.1.1    Global Adeno-Associated Virus Vector-Based Gene Therapy Market Historical Value (2016-2022) 
            6.2.1.2    Global Adeno-Associated Virus Vector-Based Gene Therapy Market Forecast Value (2023-2031)
    6.3    Global Adeno-Associated Virus Vector-Based Gene Therapy Market by Therapeutic Agents
        6.3.1    Market Overview
            6.3.1.1    Corticosteroid Drugs
            6.3.1.2    Immunosuppressive Drugs
            6.3.1.3    ACE Inhibitors and ARBs
            6.3.1.4    Others
    6.4    Global Adeno-Associated Virus Vector-Based Gene Therapy Market by Indication Type
        6.4.1    Market Overview
            6.4.1.1    Hematological Disorders
                6.4.1.1.1    Hemophilia A
                6.4.1.1.2    Hemophilia B
            6.4.1.2    Ophthalmological Disorders
                6.4.1.2.1    Choroideremia
                6.4.1.2.2    Retinitis Pigmentosa
            6.4.1.3    Lysosomal Storage Disorders
                6.4.1.3.1    Fabry Disease
                6.4.1.3.2    Pompe Disease
                6.4.1.3.3    Hunter Syndrome
            6.4.1.4    Neurological Disorders
                6.4.1.4.1    Parkinson's Disease
                6.4.1.4.2    Batten Disease
            6.4.1.5    Musculoskeletal Disorders
                6.4.1.5.1    Duchenne Muscular Dystrophy (DMD)
                6.4.1.5.2    Spinal Muscular Atrophy (SMA)
    6.5    Global Adeno-Associated Virus Vector-Based Gene Therapy Market by Treatment Channel
        6.5.1    Market Overview
            6.5.1.1    Public
            6.5.1.2    Private
    6.6    Global Adeno-Associated Virus Vector-Based Gene Therapy Market by Region
        6.6.1    Market Overview
            6.6.1.1    North America
            6.6.1.2    Europe
            6.6.1.3    Asia Pacific
            6.6.1.4    Middle East & Africa
            6.6.1.5    Latin America
        6.6.2    North America
            6.6.2.1    Historical Trend (2016-2022)
            6.6.2.2    Forecast Trend (2023-2031)
            6.6.2.3    Break Up by Country
            6.6.2.3.1    United States of America
            6.6.2.3.2    Canada
        6.6.3    Europe
            6.6.3.1    Historical Trend (2016-2022)
            6.6.3.2    Forecast Trend (2023-2031)
            6.6.3.3    Break Up by Country
                6.6.3.3.1    United Kingdom
                6.6.3.3.2    Germany
                6.6.3.3.3    France
                6.6.3.3.4    Italy
                6.6.3.3.5    Others
        6.6.4    Asia Pacific
            6.6.4.1    Historical Trend (2016-2022)
            6.6.4.2    Forecast Trend (2023-2031)
            6.6.4.3    Break Up by Country
                6.6.4.3.1    China
                6.6.4.3.2    Japan
                6.6.4.3.3    India
                6.6.4.3.4    ASEAN
                6.6.4.3.5    Australia
                6.6.4.3.6    Others
        6.6.5    Latin America
            6.6.5.1    Historical Trend (2016-2022)
            6.6.5.2    Forecast Trend (2023-2031)
            6.6.5.3    Break Up by Country
                6.6.5.3.1    Brazil
                6.6.5.3.2    Argentina
                6.6.5.3.3    Mexico
                6.6.5.3.4    Others
        6.6.6    Middle East and Africa
            6.6.6.1    Historical Trend (2016-2022)
            6.6.6.2    Forecast Trend (2023-2031)
            6.6.6.3    Break Up by Country
                6.6.6.3.1    Saudi Arabia
                6.6.6.3.2    United Arab Emirates
                6.6.6.3.3    Nigeria
                6.6.6.3.4    South Africa
                6.6.6.3.5    Others
7    Current Scenario Evaluation and Regulatory Framework 
    7.1    Emerging Therapies and Clinical Trials
    7.2    Patent Landscape 
        7.2.1    Patent Overview
            7.2.1.1    Patent Status and Expiry
            7.2.1.2    Timelines from Drug Development to Commercial Launch
            7.2.1.3    New Drug Application
                7.2.1.3.1    Documentation and Approval Process
    7.3    Cost of Treatment
    7.4    Regulatory Framework
        7.4.1    Regulatory Overview
            7.4.1.1    US FDA
            7.4.1.2    EU EMA
            7.4.1.3    INDIA CDSCO
            7.4.1.4    JAPAN PMDA
            7.4.1.5    Others
8    Challenges & Unmet Needs
    8.1    Treatment Pathway Challenges
    8.2    Compliance and Drop-Out Analysis
    8.3    Awareness and Prevention Gaps
9    Global Adeno-Associated Virus Vector-Based Gene Therapy Market Dynamics
    9.1    Market Drivers and Constraints
    9.2    SWOT Analysis
    9.3    Porter’s Five Forces Model
    9.4    Key Demand Indicators
    9.5    Key Price Indicators
    9.6    Industry Events, Initiatives & Trends 
    9.7    Value Chain Analysis
10    Supplier Landscape 
    10.1    Arya Sciences Acquisition Corp.
        10.1.1    Company Overview
        10.1.2    Product Portfolio
        10.1.3    Demographic Reach and Achievements
        10.1.4    Mergers and Acquisitions
        10.1.5    Certifications
    10.2    Pfizer Inc.
        10.2.1    Company Overview
        10.2.2    Product Portfolio
        10.2.3    Demographic Reach and Achievements
        10.2.4    Mergers and Acquisitions
        10.2.5    Certifications
    10.3    Roche Holding AG
        10.3.1    Company Overview
        10.3.2    Product Portfolio
        10.3.3    Demographic Reach and Achievements
        10.3.4    Mergers and Acquisitions
        10.3.5    Certifications
    10.4    BioMarin Pharmaceutical Inc.
        10.4.1    Company Overview
        10.4.2    Product Portfolio
        10.4.3    Demographic Reach and Achievements
        10.4.4    Mergers and Acquisitions
        10.4.5    Certifications
    10.5    Bayer AG
        10.5.1    Company Overview
        10.5.2    Product Portfolio
        10.5.3    Demographic Reach and Achievements
        10.5.4    Mergers and Acquisitions
        10.5.5    Certifications
    10.6    Coave Therapeutics
        10.6.1    Company Overview
        10.6.2    Product Portfolio
        10.6.3    Demographic Reach and Achievements
        10.6.4    Mergers and Acquisitions
        10.6.5    Certifications
    10.7    MeiraGTx Limited
        10.7.1    Company Overview
        10.7.2    Product Portfolio
        10.7.3    Demographic Reach and Achievements
        10.7.4    Mergers and Acquisitions
        10.7.5    Certifications
    10.8    Neurocrine Biosciences, Inc.
        10.8.1    Company Overview
        10.8.2    Product Portfolio
        10.8.3    Demographic Reach and Achievements
        10.8.4    Mergers and Acquisitions
        10.8.5    Certifications
    10.9    Biogen, Inc.
        10.9.1    Company Overview
        10.9.2    Product Portfolio
        10.9.3    Demographic Reach and Achievements
        10.9.4    Mergers and Acquisitions
        10.9.5    Certifications
    10.10    Sangamo Therapeutics
        10.10.1    Company Overview
        10.10.2    Product Portfolio
        10.10.3    Demographic Reach and Achievements
        10.10.4    Mergers and Acquisitions
        10.10.5    Certifications
    10.11    Sarepta Therapeutics, Inc.
        10.11.1    Company Overview
        10.11.2    Product Portfolio
        10.11.3    Demographic Reach and Achievements
        10.11.4    Mergers and Acquisitions
        10.11.5    Certifications
11    Recommendations and Discussion 
12    Global Adeno-Associated Virus Vector-Based Gene Therapy Drugs Distribution Model (Additional Insight)

    12.1    Overview
    12.2    Potential Distributors
    12.3    Key Parameters for Distribution Partner Assessment
13    Payment Methods (Additional Insight)
    13.1    Government Funded
    13.2    Private Insurance
    13.3    Out-of-Pocket

 

*Additional insights provided are customisable as per client requirements.

Key Questions Answered in the Report

Adeno-associated virus (AAV) is a group of viruses infecting humans and some other species. Adenovirus belongs to the family Parvoviridae. These viruses do not cause any disease but show a mild immune response.

The AAV vectors are mainly used as drug delivery systems for transporting the required therapeutic product at the target site.

The different regions in the adeno-associated virus vector-based gene therapy market are North America, Europe, Asia Pacific, Latin America and Middle East and Africa.

Based on the therapeutic agents, the market are used in corticosteroid drugs, immunosuppressive drugs, ACE inhibitors and ARBs, among others.

The treatment channels in the market are public and private.

Advancements in the biotechnological sector, rising expenditure in the R&D sector, effective treatment and increasing key players are driving the growth of the market.

North America is anticipated to hold a significant share of the market during the forecast period.

The key companies involved in the market are Arya Sciences Acquisition Corp, Pfizer Inc., Roche Holding AG, BioMarin Pharmaceutical Inc., Bayer AG, Coave Therapeutics, MeiraGTx Limited, Neurocrine Biosciences, Inc., Biogen, Inc., Sangamo Therapeutics, and Sarepta Therapeutics, Inc., among others.

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