Report cover image

AAV Gene Therapy Market

Published Dec 19, 2025
Length 168 Pages
SKU # CMI20729628

Description

The Global AAV Gene Therapy Market is estimated to be valued at USD 3.46 Bn in 2025 and is expected to reach USD 16.97 Bn by 2032, growing at a compound annual growth rate (CAGR) of 25.5% from 2025 to 2032. The global AAV gene therapy market represents a revolutionary frontier in modern medicine, utilizing Adeno-Associated Virus (AAV) vectors as sophisticated delivery systems to transport therapeutic genes directly into target cells for treating various genetic disorders and acquired diseases.

AAV gene therapy has emerged as a groundbreaking therapeutic approach due to its exceptional safety profile, minimal immunogenicity, and ability to achieve long-lasting gene expression without integrating into the host genome.

This innovative treatment modality leverages the unique characteristics of AAV vectors, which are naturally occurring, non-pathogenic viruses that have been extensively engineered to remove their replicative capacity while retaining their remarkable ability to transduce both dividing and non-dividing cells across multiple tissue types.

The market encompasses a diverse range of therapeutic applications including inherited retinal diseases, hemophilia, spinal muscular atrophy, and various central nervous system disorders, with numerous clinical trials demonstrating promising efficacy outcomes. As regulatory agencies worldwide continue to approve AAV-based gene therapies and pharmaceutical companies intensify their research and development efforts, the market is experiencing unprecedented growth driven by technological advancements in vector engineering, improved manufacturing processes, and expanding therapeutic targets that collectively position AAV gene therapy as a transformative solution for previously untreatable genetic conditions.

Market Dynamics

The global AAV gene therapy market is propelled by several compelling drivers that collectively fuel its robust expansion across the healthcare landscape. The primary growth catalyst stems from the increasing prevalence of genetic disorders and rare diseases worldwide, coupled with the urgent unmet medical need for effective treatments in conditions previously deemed incurable, creating substantial market demand for innovative AAV-based therapeutic solutions.

Significant technological advancements in vector engineering, including the development of novel AAV capsids with enhanced tissue specificity and improved transduction efficiency, have dramatically expanded the therapeutic potential and clinical applicability of these treatments. Supportive regulatory frameworks established by agencies such as the U.S. FDA and EMA, including expedited approval pathways for rare disease treatments and orphan drug designations, have accelerated the development timeline and market entry for AAV gene therapies.

However, the market faces notable restraints primarily centered around the exceptionally high manufacturing costs associated with AAV vector production, which translates into substantial treatment expenses that limit patient accessibility and create reimbursement challenges for healthcare systems globally.

Additionally, potential safety concerns including immunogenic responses to AAV vectors and the risk of genotoxicity continue to pose regulatory hurdles that may delay product approvals and market penetration. Despite these challenges, the market presents tremendous opportunities driven by expanding therapeutic applications beyond rare genetic diseases into more prevalent conditions such as cardiovascular diseases, neurodegenerative disorders, and ophthalmologic conditions, while emerging manufacturing technologies including suspension cell culture systems and novel purification methods promise to reduce production costs and improve scalability, ultimately enhancing market accessibility and growth potential.

Key Features of the Study
  • This report provides in-depth analysis of the global AAV gene therapy market, and provides market size (USD Billion) and compound annual growth rate (CAGR%) for the forecast period (2025–2032), considering 2024 as the base year
  • It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market
  • This study also provides key insights about market drivers, restraints, opportunities, new product launches or approvals, market trends, regional outlook, and competitive strategies adopted by key players
  • It profiles key players in the global AAV gene therapy market based on the following parameters – company highlights, products portfolio, key highlights, financial performance, and strategies
  • Key companies covered as a part of this study include Novartis (AveXis), Spark Therapeutics (Roche), BioMarin Pharmaceutical, Sarepta Therapeutics, uniQure, Regenxbio, Audentes Therapeutics (Astellas), Voyager Therapeutics, MeiraGTx, Asklepios BioPharmaceutical (AskBio – Bayer), Krystal Biotech, Genethon, BridgeBio Gene Therapy, Passage Bio, and Solid Biosciences
  • Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
  • The global AAV gene therapy market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
  • Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global AAV gene therapy market
Market Segmentation
  • Vector Serotype Insights (Revenue, USD Bn, 2020 - 2032)
  • AAV9
  • AAV2
  • AAV5
  • AAV8
  • AAV1
  • Engineered/Synthetic AAV Variants
  • Therapeutic Area Insights (Revenue, USD Bn, 2020 - 2032)
  • Neurological Disorders
  • Ophthalmic Disorders
  • Rare Genetic Disorders
  • Hematological Disorders
  • Muscular Disorders
  • Metabolic Disorders
  • Cardiovascular Disorders
  • Oncology
  • Others
  • Target Tissue Insights (Revenue, USD Bn, 2020 - 2032)
  • Central Nervous System
  • Eye/Retina
  • Liver
  • Muscle
  • Lung
  • Heart
  • Blood/Bone Marrow
  • Route of Administration Insights (Revenue, USD Bn, 2020 - 2032)
  • Intravenous
  • Intrathecal
  • Intracerebral
  • Subretinal
  • Intravitreal
  • Intramuscular
  • Intracardiac
  • Indication Type Insights (Revenue, USD Bn, 2020 - 2032)
  • Monogenic Disorders
  • Polygenic Disorders
  • Gene Payload Type Insights (Revenue, USD Bn, 2020 - 2032)
  • Gene Replacement
  • Gene Silencing
  • Gene Augmentation
  • Development Stage Insights (Revenue, USD Bn, 2020 - 2032)
  • Preclinical
  • Phase I
  • Phase II
  • Phase III
  • Manufacturing Type Insights (Revenue, USD Bn, 2020 - 2032)
  • In-house Manufacturing
  • Contract Manufacturing (CDMO/CMO)
  • End User Insights (Revenue, USD Bn, 2020 - 2032)
  • Biopharmaceutical Companies
  • Research Institutes and Academic Centers
  • Hospitals and Specialty Clinics
  • Others (CROs)
  • Regional Insights (Revenue, USD Bn, 2020 - 2032)
  • North America
  • U.S.
  • Canada
  • Latin America
  • Brazil
  • Argentina
  • Mexico
  • Rest of Latin America
  • Europe
  • Germany
  • U.K.
  • Spain
  • France
  • Italy
  • Russia
  • Rest of Europe
  • Asia Pacific
  • China
  • India
  • Japan
  • Australia
  • South Korea
  • ASEAN
  • Rest of Asia Pacific
  • Middle East
  • GCC Countries
  • Israel
  • Rest of Middle East
  • Africa
  • South Africa
  • North Africa
  • Central Africa
  • Key Players Insights
  • Novartis (AveXis)
  • Spark Therapeutics (Roche)
  • BioMarin Pharmaceutical
  • Sarepta Therapeutics
  • uniQure
  • Regenxbio
  • Audentes Therapeutics (Astellas)
  • Voyager Therapeutics
  • MeiraGTx
  • Asklepios BioPharmaceutical (AskBio – Bayer)
  • Krystal Biotech
  • Genethon
  • BridgeBio Gene Therapy
  • Passage Bio
  • Solid Biosciences

Table of Contents

168 Pages
1. Research Objectives and Assumptions
Research Objectives
Assumptions
Abbreviations
2. Market Purview
Report Description
Market Definition and Scope
Executive Summary
Global AAV Gene Therapy Market, By Vector Serotype
Global AAV Gene Therapy Market, By Therapeutic Area
Global AAV Gene Therapy Market, By Target Tissue
Global AAV Gene Therapy Market, By Route of Administration
Global AAV Gene Therapy Market, By Indication Type
Global AAV Gene Therapy Market, By Gene Payload Type
Global AAV Gene Therapy Market, By Development Stage
Global AAV Gene Therapy Market, By Manufacturing Type
Global AAV Gene Therapy Market, By End User
Global AAV Gene Therapy Market, By Region
3. Market Dynamics, Regulations, and Trends Analysis
Market Dynamics
Driver
Restraint
Opportunity
Impact Analysis
Key Developments
Regulatory Scenario
Product Launches/Approvals
PEST Analysis
PORTER’s Analysis
Merger and Acquisition Scenario
Industry Trends
4. Global AAV Gene Therapy Market, By Vector Serotype, 2020-2032, (USD Bn)
Introduction
Market Share Analysis, 2025 and 2032 (%)
Y-o-Y Growth Analysis, 2021 - 2032
Segment Trends
AAV9
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
AAV2
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
AAV5
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
AAV8
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
AAV1
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Engineered/Synthetic AAV Variants
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
5. Global AAV Gene Therapy Market, By Therapeutic Area, 2020-2032, (USD Bn)
Introduction
Market Share Analysis, 2025 and 2032 (%)
Y-o-Y Growth Analysis, 2021 - 2032
Segment Trends
Neurological Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Ophthalmic Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Rare Genetic Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Hematological Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Muscular Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Metabolic Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Cardiovascular Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Oncology
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Others
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
6. Global AAV Gene Therapy Market, By Target Tissue, 2020-2032, (USD Bn)
Introduction
Market Share Analysis, 2025 and 2032 (%)
Y-o-Y Growth Analysis, 2021 - 2032
Segment Trends
Central Nervous System
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Eye/Retina
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Liver
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Muscle
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Lung
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Heart
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Blood/Bone Marrow
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
7. Global AAV Gene Therapy Market, By Route of Administration, 2020-2032, (USD Bn)
Introduction
Market Share Analysis, 2025 and 2032 (%)
Y-o-Y Growth Analysis, 2021 - 2032
Segment Trends
Intravenous
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Intrathecal
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Intracerebral
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Subretinal
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Intravitreal
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Intramuscular
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Intracardiac
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
8. Global AAV Gene Therapy Market, By Indication Type, 2020-2032, (USD Bn)
Introduction
Market Share Analysis, 2025 and 2032 (%)
Y-o-Y Growth Analysis, 2021 - 2032
Segment Trends
Monogenic Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Polygenic Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
9. Global AAV Gene Therapy Market, By Gene Payload Type, 2020-2032, (USD Bn)
Introduction
Market Share Analysis, 2025 and 2032 (%)
Y-o-Y Growth Analysis, 2021 - 2032
Segment Trends
Gene Replacement
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Gene Silencing
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Gene Augmentation
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
10. Global AAV Gene Therapy Market, By Development Stage, 2020-2032, (USD Bn)
Introduction
Market Share Analysis, 2025 and 2032 (%)
Y-o-Y Growth Analysis, 2021 - 2032
Segment Trends
Preclinical
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Phase I
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Phase II
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Phase III
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
11. Global AAV Gene Therapy Market, By Manufacturing Type, 2020-2032, (USD Bn)
Introduction
Market Share Analysis, 2025 and 2032 (%)
Y-o-Y Growth Analysis, 2021 - 2032
Segment Trends
In-house Manufacturing
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Contract Manufacturing (CDMO/CMO)
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
12. Global AAV Gene Therapy Market, By End User, 2020-2032, (USD Bn)
Introduction
Market Share Analysis, 2025 and 2032 (%)
Y-o-Y Growth Analysis, 2021 - 2032
Segment Trends
Biopharmaceutical Companies
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Research Institutes and Academic Centers
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Hospitals and Specialty Clinics
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
Others (CROs)
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (USD Bn)
13. Global AAV Gene Therapy Market, By Region, 2020 - 2032, Value (USD Bn)
Introduction
Market Share (%) Analysis, 2025, 2028 & 2032, Value (USD Bn)
Market Y-o-Y Growth Analysis (%), 2021 - 2032, Value (USD Bn)
Regional Trends
North America
Introduction
Market Size and Forecast, By Vector Serotype, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Therapeutic Area, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Target Tissue, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Route of Administration, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Indication Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Gene Payload Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Development Stage, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Manufacturing Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By End User, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Country, 2020 - 2032, Value (USD Bn)
U.S.
Canada
Latin America
Introduction
Market Size and Forecast, By Vector Serotype, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Therapeutic Area, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Target Tissue, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Route of Administration, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Indication Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Gene Payload Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Development Stage, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Manufacturing Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By End User, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Country, 2020 - 2032, Value (USD Bn)
Brazil
Argentina
Mexico
Rest of Latin America
Europe
Introduction
Market Size and Forecast, By Vector Serotype, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Therapeutic Area, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Target Tissue, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Route of Administration, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Indication Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Gene Payload Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Development Stage, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Manufacturing Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By End User, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Country, 2020 - 2032, Value (USD Bn)
Germany
U.K.
Spain
France
Italy
Russia
Rest of Europe
Asia Pacific
Introduction
Market Size and Forecast, By Vector Serotype, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Therapeutic Area, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Target Tissue, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Route of Administration, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Indication Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Gene Payload Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Development Stage, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Manufacturing Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By End User, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Country, 2020 - 2032, Value (USD Bn)
China
India
Japan
Australia
South Korea
ASEAN
Rest of Asia Pacific
Middle East
Introduction
Market Size and Forecast, By Vector Serotype, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Therapeutic Area, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Target Tissue, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Route of Administration, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Indication Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Gene Payload Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Development Stage, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Manufacturing Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By End User, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Country, 2020 - 2032, Value (USD Bn)
GCC Countries
Israel
Rest of Middle East
Africa
Introduction
Market Size and Forecast, By Vector Serotype, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Therapeutic Area, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Target Tissue, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Route of Administration, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Indication Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Gene Payload Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Development Stage, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Manufacturing Type, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By End User, 2020 - 2032, Value (USD Bn)
Market Size and Forecast, By Country/Region, 2020 - 2032, Value (USD Bn)
South Africa
North Africa
Central Africa
14. Competitive Landscape
Novartis (AveXis)
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
Spark Therapeutics (Roche)
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
BioMarin Pharmaceutical
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
Sarepta Therapeutics
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
uniQure
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
Regenxbio
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
Audentes Therapeutics (Astellas)
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
Voyager Therapeutics
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
MeiraGTx
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
Asklepios BioPharmaceutical (AskBio – Bayer)
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
Krystal Biotech
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
Genethon
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
BridgeBio Gene Therapy
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
Passage Bio
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
Solid Biosciences
Company Highlights
Product Portfolio
Key Developments
Financial Performance
Strategies
15. Analyst Recommendations
Wheel of Fortune
Analyst View
Coherent Opportunity Map
16. References and Research Methodology
References
Research Methodology
About us
*Browse 32 market data tables and 28 figures on ‘AAV Gene Therapy Market' - Global forecast to 2032
How Do Licenses Work?
Request A Sample
Head shot

Questions or Comments?

Our team has the ability to search within reports to verify it suits your needs. We can also help maximize your budget by finding sections of reports you can purchase.