The adeno-associated virus (AAV) vector-based gene therapy market reached USD 5.72 billion in 2024 and is projected to grow at a CAGR of 21.30%, reaching USD 39.45 billion by 2034. Market expansion is primarily driven by technological advancements in gene therapy, growing investment in research for rare and inherited genetic disorders, and the increasing approval of AAV-based therapies for various conditions.

Rising prevalence of genetic and chronic diseases, coupled with enhanced clinical trial pipelines and government support for innovative therapies, further fuels market growth. Additionally, the integration of AAV vectors in personalized medicine is expanding therapeutic potential, particularly for conditions that currently lack effective treatments.

Key Market Segmentation

By Therapeutic Application:

Neurological Disorders

Ophthalmic Disorders

Muscular Disorders

Hematological Disorders

Metabolic Disorders

By Target Disease:

Rare Diseases

Cancer

Inherited Genetic Disorders

By End User:

Hospitals and Clinics

Research Institutes and Universities

Biotechnology and Pharmaceutical Companies

By Region:

North America

Europe

Asia Pacific

Latin America

Middle East and Africa

Leading Companies in the AAV Vector-Based Gene Therapy Market

Major players innovating and expanding the market include:

• Arya Sciences Acquisition Corp.

• Pfizer Inc.

• BioMarin Pharmaceutical Inc.

• Bayer AG

• Coave Therapeutics

• MeiraGTx Limited

• Neurocrine Biosciences, Inc.

• Biogen, Inc.

• Sangamo Therapeutics

• Sarepta Therapeutics, Inc.

These companies focus on developing advanced vector platforms, improving delivery efficiency, and expanding therapeutic indications to strengthen their market position globally.

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Trends and Opportunities Shaping the Market

Expansion in Rare and Inherited Disease Treatment: High unmet needs in rare genetic disorders drive clinical development and commercial opportunities.

Technological Innovations: Improved AAV vector design and manufacturing processes enhance safety, targeting efficiency, and therapeutic efficacy.

Collaborations and Partnerships: Biotech-pharma partnerships accelerate research, clinical trials, and product commercialization.

Growing Clinical Trials: Increased number of global trials targeting neurological, ophthalmic, and muscular disorders.

Emerging Markets Potential: Rising investment in healthcare infrastructure and adoption of advanced therapies in Asia Pacific and Latin America.

The AAV vector-based gene therapy market is poised for substantial growth through 2034, driven by cutting-edge gene therapy technologies, rising disease prevalence, and strong investment in innovative solutions, positioning it as a critical segment in modern healthcare and personalized medicine.