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Adeno Associated Virus (AAV) Vectors in Gene Therapy Market Research Report

Published: 2025 - January

Report Code: VMR-17689

Region: Global

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Adeno Associated Virus (AAV) Vectors in Gene Therapy Market Analysis

The Adeno Associated Virus (AAV) Vectors in Gene Therapy Market represents a transformative segment of the biopharmaceutical industry, focusing on the development and application of AAV-based vectors for delivering therapeutic genes. These vectors are highly efficient, non-pathogenic delivery systems tailored to treat genetic, neurological, ocular, and metabolic disorders. With their specificity, long-term gene expression, and reduced immunogenicity, AAV vectors are central to advancing personalized and curative therapies.

Disruptive Impact and Opportunities:

Emerging therapies like RGX-314 and DTX401 leverage novel capsid designs and targeted delivery, redefining treatment paradigms for diseases like hemophilia and retinal disorders. Simplified dosing regimens through innovative vectors, such as AMT-061, enable broader patient accessibility. Proven safety profiles, as seen with Voretigene neparvovec and Onasemnogene abeparvovec, highlight the minimal risks associated with AAV vectors. With expanding applications in untapped indications, including neurology and rare genetic disorders, the market holds significant growth potential.

Adeno Associated Virus (AAV) Vectors in Gene Therapy Market Segmentation - Emerging Drugs

  • GS010

  • DTX401

  • AAV5-RPGR

  • AMT-061

  • RGX-314

Adeno Associated Virus (AAV) Vectors in Gene Therapy Market Segmentation - Marketed Drugs

  • Voretigene neparvovec

  • Onasemnogene abeparvovec

  • Alipogene tiparvovec

  • Timrepigene emparvovec

  • Valoctocogene roxaparvovec

  • Giroctocogene fitelparvovec

Key Companies:

  • Hoffmann-La Roche

  • Novartis AG

  • UniQure

  • Biogen

  • Biomarin Pharmaceutical

  • Pfizer

  • Neurophth Therapeutics

  • Adrenas Therapeutics

  • Amicus Therapeutics

Adeno Associated Virus (AAV) Vectors in Gene Therapy Market Segmentation -  By Type

·  Natural AAV Vectors

  • AAV1

  • AAV2

  • AAV5

  • AAV6

  • AAV8

  • AAV9

  • Others

·  Engineered/Modified AAV Vectors

  • Self-Complementary AAV (scAAV)

  • Recombinant AAV (rAAV)

  • Synthetic AAV Capsid Variants

Adeno Associated Virus (AAV) Vectors in Gene Therapy Market Segmentation -  By Administration Type

·  In Vivo Administration

  • Intravenous (IV)

  • Intramuscular (IM)

  • Intrathecal

  • Subretinal

  • Others

·  Ex Vivo Administration

  • Hematopoietic Stem Cells (HSCs)

  • T Cells

  • Other Cell Types

What’s in It for You?

  • Comprehensive insights into emerging and marketed therapies driving innovation.

  • Strategic analysis of key players shaping the AAV vectors market.

  • Actionable intelligence on market opportunities in rare and genetic diseases.

  • Data-driven recommendations for investment, partnership, and R&D strategies.

  1. Adeno Associated Virus Vectors In Gene Therapy Market - Executive Summary

    1. Introduction

    2. Objectives

    3. Key Findings

      1. Market Size 2025 & 2030:: By Key Country (10MM)

      2. Global Market Size 2025 & 2030:: By Key Segment

      3. Key Investments & Startup Analysis

    4. Research Methodology

  2. Understanding the Disease

    1. Disease Overview

    2. Classification

    3. Signs and Symptoms

    4. Risk Factors

    5. Causes

    6. Disease Biology & Digital Innovations

    7. Stages & Staging System

    8. Diagnostic Algorithm

    9. Current Treatment Practices & Algorithm

    10. Current Standard of Care and Treatment Gaps

    11. Patient Demographics and Treatment Pathways

  3. Guidelines

  4. Unmet Needs

  5. Epidemiology and Patient Population

    1. Epidemiology Key Findings

    2. Assumptions and Rationale: 10MM

    3. Epidemiology Scenario: 10MM

    4. U.S. Epidemiology Scenario

    5. EU-5 Epidemiology

      1. U.K. Epidemiology Scenario

      2. Germany Epidemiology Scenario

      3. France Epidemiology Scenario

      4. Italy Epidemiology Scenario

      5. Spain Epidemiology Scenario

    6. Japan Epidemiology Scenario

    7. China Epidemiology Scenario

    8. Australia Epidemiology Scenario

    9. India Epidemiology Scenario   

  6. Real-world Data & Real-world Evidence

  7. Drug Development Landscape

    1. Existing Key Drug Candidate Profiles/ Marketed Therapies

    2. Competitive Analysis and Differentiation

    3. Overview of Similar/Competing Drugs in Clinical Trials

    4. Future Trends and Emerging Drugs

  8. Regulatory Strategy and Potential Challenges

    1. Regulatory Pathways in Key Markets

    2. Anticipated Regulatory Hurdles and Mitigation Strategies

    3. Case Studies in Oncology Drug Regulation

    4. Impact of Potential Changes to Regulatory Framework

  9. Commercial Landscape

    1. Market Size & Growth Rates

    2. Key Approvals & Anticipated Loss of Exclusivity

    3. PESTLE & Porter’s Five Forces Analysis

    4. Market Shares, Positioning/Ranking

    5. Market Drivers

    6. Identification of Threats

    7. Digital Evolution in Commercialization

  10. Market Segmentation

  11. Pricing, Reimbursement, and Access

    1. Competitive Pricing Analysis

    2. Reimbursement Landscape and Challenges

    3. Strategies for Market Access and Equity

    4. Patient Spending/Expenditure Analysis

  12. Future Trends, Disruptions, and Opportunities

    1. Analysis of Emerging Trends

    2. Technological Impact

    3. Impact of Potential Market Disruptors

    4. Opportunities for Future Development and Expansion

    5. Considerations for Investment Opportunities

  13. Global Market Dynamics

    1. Regional Regulatory Disparities

    2. Cross-Border Partnership Strategies

    3. Global Supply Chain Dynamics

    4. Case Studies: Success and Failure in Global Markets

    5. Strategies for Global Expansion and Localization

  14. Company Profiles

 

 

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