Adeno-associated Virus (AAV) Vector-based Generket size to Reach USD 32.96 Bn by 2034 Therapy Ma

Vantage Market Research releases its latest comprehensive report on the ‘Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market’ with a forecast period of 2025-2034. In 2023, the global Adeno-Associated Virus (AAV) vector-based gene therapy market size was USD 5.1 billion, and is calculated at USD 6.02 billion in 2024. The Adeno-Associated Virus (AAV) vector-based gene therapy market is projected to reach USD 32.96  billion in 2034, and register a revenue CAGR of 18.2% over the forecast period (2025-2034).

Market Overview:

The global Adeno-Associated Virus (AAV) vector-based gene therapy market is expected to continue to register a robust revenue growth rate over the forecast period, supported to a major extent by key factors such as increasing prevalence of genetic and rare diseases, advances in biotechnology, and rising investment in Research and Development (R&D) and healthcare infrastructure worldwide. Conditions such as Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA), and hemophilia are now actively targeted by AAV-based therapies, which provide long-term solutions with minimized side effects compared to conventional treatments. Also, continued advancements in AAV vector technology have led to more effective and precisely targeted gene therapies, contributing to higher success rates in clinical trials. 

Moreover, favorable regulatory environments, particularly in North America and Europe, are accelerating approvals for gene therapies and research into Parkinson’s disease (PD) and Alzheimer’s disease (AD), metabolic disorders such as lysosomal storage disorders, and even cancers. Supportive frameworks by agencies like the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are speeding-up pathways for new treatments, thereby enhancing the ability of pharmaceutical companies to launch innovative solutions. In addition, strategic partnerships and mergers between biopharmaceutical companies, research institutions, and technology providers are driving rapid development and commercialization in the AAV gene therapy landscape.

AAV vector-based gene therapy remains highly attractive due to its low immunogenicity, high efficiency, and potential for a one-time administration with sustained effects. Applications for these therapies extend beyond genetic and rare diseases to include promising uses in cardiovascular and metabolic conditions, further expanding market potential. Recent trends indicate a surge in investment across emerging markets such as the Asia Pacific, strengthening access to advanced treatments globally. Leading companies like Pfizer and Sarepta Therapeutics continue to develop and expand product pipelines, supporting the growth of AAV-based therapies and catering to increasing demand in the market.

Some Key Takeaways:

• North America Adeno-Associated Virus (AAV) vector-based gene therapy market continued to lead in terms of revenue share. Factors such as presence of advanced healthcare infrastructure, strong regulatory support, and significant investments in biotechnology. 
• The US in particular, is at the forefront, with major pharmaceutical and biotech companies such as Pfizer, Sarepta Therapeutics, and Spark Therapeutics based in the country.
• In Europe, Germany, France, and the UK are leading country-level markets, supported by the European Union regulatory frameworks facilitating faster approval processes for gene therapies.
• In the Asia Pacific market, Japan and China are leading markets due to increasing healthcare expenditure and rising investments in gene therapy.
• Japan has a particularly strong regulatory system that encourages biotech innovation, and Sangamo Therapeutics and Regenxbio are targeting these regions for expanding their gene therapy pipelines.
• The neurological disorders segment is expected to account for largest revenue share among the disease type segments in the adeno-associated virus vector-based gene therapy market over the forecast period.
• The AAV9 segment among the vector serotype segments is expected to account for largest revenue share over the forecast period.
• The intravenous (IV) administration segment is expected to account for largest share among the administration route segments over the forecast period. 
• The biopharmaceutical companies segment is expected to account for largest revenue share among the end user segments in the market over the forecast period. 
• The competitive landscape of the global Adeno-Associated Virus (AAV) vector-based gene therapy market is highly dynamic, with a number of major players competing for market share. 
• Major companies are advancing innovative therapies and expanding their global reach, with Pfizer, Sarepta Therapeutics, BioMarin Pharmaceutical, and UniQure maintaining significant dominance.

Company List:

• Pfizer
• BioMarin Pharmaceutical
• Sarepta Therapeutics
• Takeda
• UniQure
• Regenxbio
• Adverum Biotechnologies
• Spark Therapeutics (Roche)
• Astellas Pharma
• Bayer
• Ultragenyx Pharmaceutical
• Audentes Therapeutics (Astellas)
• Homology Medicines
• GenSight Biologics
• Sangamo Therapeutics 

Recent Developments:

• October 24, 2024: Dyno Therapeutics, Inc., which is a genetic technologies company leveraging Artificial Intelligence (AI) to advance in vivo gene delivery, announced its second research collaboration with Roche. The objective of this partnership is to create next-generation adeno-associated virus vectors designed for gene therapies addressing neurological disorders. This collaboration builds on an initial agreement formed in October 2020, which focused on AAV vectors for neurological and liver-targeted therapies. With this latest agreement, Dyno will grant Roche extended access to its advanced platform and sequence design technologies, which enhance in vivo gene delivery capabilities and aim to bring innovative solutions to the gene therapy landscape.
• July 11, 2024: Merck signed a non-binding Memorandum of Understanding (MoU) with Gene Therapy Research Institution Co., Ltd. (GTRI), which is a Japanese biotech specializing in adeno-associated virus (AAV) gene therapies. GTRI will use Merck’s Sf-RVN Insect Cell Line platform for GMP production of its AAV-based gene therapy targeting Parkinson’s Disease. Under the MoU, Merck will support GTRI in building a scalable AAV production platform and provide technical assistance, training, and process optimization at its M Lab Collaboration Center in Tokyo. This partnership leverages Merck’s cell line technology to advance GTRI’s gene therapy development and production capabilities.

Global Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Segmentation:

By Disease Type:

• Hematological Malignancies
• Neurological Disorders
• Cardiovascular Diseases
• Ophthalmic Diseases
• Oncology

By Vector Serotype:

• AAV1
• AAV2
• AAV5
• AAV6
• AAV8
• AAV9

By Administration Route:

• Intravenous
• Intramuscular
• Subcutaneous
• Intraocular
• Intracerebral

By End User:

• Hospitals
• Research Institutes
• Biopharmaceutical Companies
  • United States

Regional Segmentation: 

North America

• Canada
• Mexico

Europe

• Germany
• United Kingdom
• France
• Italy
• Spain
• Rest of Europe

Asia Pacific

• China
• Japan
• India
• Rest of Asia Pacific

Latin America

• Brazil
• Argentina
• Rest of Latin America 

Middle East & Africa

• Saudi Arabia
• South Africa
• United Arab Emirates
• Israel
• Rest of MEA