Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Report, By Gene Therapy Application (Neurological Disorders, Ophthalmic Diseases, Muscular Disorders, Hematological Disorders); AAV Serotype (AAV1, AAV2, AAV5, AAV9); End-User (Hospitals, Research Institutes, Biopharmaceutical Companies); Therapeutic Area (Rare Diseases, Oncology, Cardiovascular Diseases, Genetic Disorders); and Regions 2024-2032

Market Overview:

"The global Adeno-Associated Virus (AAV) vector-based gene therapy market size reached US$ 5.2 billion in 2023. Looking forward, Reports and Insights expects the market to reach US$ 18.4 billion in 2032, exhibiting a growth rate (CAGR) of 15.1% during 2024-2032."

Report Attributes	Details
Base Year	2023
Forecast Years	2024-2032
Historical Years	2021-2023
Market Growth Rate (2024-2032)	15.1%

Adeno-Associated Virus (AAV) vectors are a type of viral vector commonly used in gene therapy. It is a small, non-pathogenic, and non-enveloped virus that belongs to the Parvoviridae family, it does not cause human disease, and its genome is a single-stranded DNA. There are currently two classes of recombinant AAVs (rAAVs) in use, including self-complementary AAV (scAAV) and single-stranded AAV (ssAAV). The rAAV gene therapy comprises gene silencing, gene replacement, gene addition, and gene editing.

In the context of gene therapy, the AAV genome is modified by removing its viral genes and replacing them with the therapeutic gene of interest. This modified AAV is then used as a vector to transport the therapeutic gene into the patient's cells. Adeno-associated virus vectors have emerged as pivotal tools in gene therapy, driving advancements in the treatment of genetic disorders. Leveraging AAV's low immunogenicity, persistent gene expression, and broad tropism, researchers are developing innovative technologies and solutions to address various genetic diseases. The benefits of AAV vectors, such as their safety profile and ability to facilitate long-term gene expression, contribute to their increasing adoption.

Some major trends in the market include the exploration of novel AAV serotypes, enhancing cargo capacity, and optimizing vector production methods. Also, collaborations between pharmaceutical companies and research institutions are driving key developments in the sector. As the field continues to evolve, the focus on refining AAV vector technologies and expanding their therapeutic applications positions them at the forefront of the gene therapy landscape, offering hope for patients with genetic disorders.

Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Trends and Drivers:

AAV vectors exhibit a favorable safety profile, with low immunogenicity, reducing the risk of adverse immune responses in patients. The persistent and long-term gene expression facilitated by AAV vectors enhances the efficacy of gene therapies, providing sustained therapeutic benefits. The broad tropism of AAV allows for the targeting of various cell types and tissues, expanding the potential applications of gene therapy across diverse genetic disorders. In addition, the versatility of AAV vectors in delivering therapeutic genes contributes to their widespread adoption, offering a platform for addressing a range of genetic diseases. Increasing prevalence of genetic conditions is also driving need for gene therapy treatments, and AAV vectors have demonstrated potential in the treatment of a number of hereditary disorders. Awareness regarding the potential advantages of gene therapy has been resulting in increasing acceptability Another major factor supporting steady progress are research and development initiatives in the field of gene therapy and favorable investment and funding from the public and commercial sectors.

Moreover, increasing number of clinical trials and research activities in the gene therapy space is supporting growth of the market to a major extent. Pharmaceutical companies and research institutions are increasingly exploring AAV vectors, fostering collaborations that drive innovation and advance the development of new treatments. Continuous and ongoing developments in the field is also enabling better understanding of genetic causes of certain disorders and AAV vectors are aiding in developing specific and more potent gene therapies. AAV vector-based gene treatments have been gaining credibility owing to positive outcomes of clinical trials and regulatory approvals for medicines.

These factors collectively contribute to the positive trajectory of the global Adeno-Associated Virus vector-based gene therapy market, as the field continues to witness technological advancements and therapeutic breakthroughs, offering hope for patients with previously incurable genetic conditions.

Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Restraining Factors:

The limited cargo capacity of AAV vectors, restricting the size of therapeutic genes that can be delivered is a major factor restricting wider adoption. Pre-existing immunity in the population due to natural AAV infections poses another hurdle, potentially hindering the effectiveness of gene therapy. Also, the potential for off-target effects and insertional mutagenesis raises safety concerns.

High manufacturing costs and complexities associated with large-scale production of AAV vectors also have an impact on wider use. Broad accessibility of AAV-based gene therapies can also be impacted by scalability issues, and inconsistency with regard to pricing and manufacturing. Moreover, regulatory challenges and uncertainties surrounding long-term safety data present hurdles for market approval. There is also the threat of alternatives and non-viral techniques and lentiviral vectors are alternative gene delivery technologies to consider.

These constraints, coupled with negative trends such as slower-than-expected clinical trial progress and scalability issues, contribute to challenges in realizing the full revenue potential of the global Adeno-Associated virus vector-based gene therapy market.

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

Increasing focus on developing novel AAV serotypes with improved transduction efficiency and tissue specificity presents an avenue for differentiation and competitive advantage. Collaborations with research institutions and academic partners can facilitate the exploration of innovative applications and expand the therapeutic scope of AAV vectors, offering new revenue streams. Also, advancements in vector engineering technologies that enhance cargo capacity and allow for the delivery of larger therapeutic genes create opportunities for expanding the applicability of AAV-based gene therapies.

As regulatory agencies demonstrate rising interest in gene therapy, companies can leverage a proactive approach to navigate regulatory landscapes, ensuring smoother market entry and approval processes. Furthermore, strategic investments in scalable and cost-effective vector production methods can address manufacturing challenges, positioning companies to meet the increasing demand for AAV vectors in gene therapy.

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

By Gene Therapy Application

• Neurological Disorders
• Ophthalmic Diseases
• Muscular Disorders
• Hematological Disorders

The neurological disorders segment is expected to account for largest revenue share over the forecast period. This can be attributed to increasing prevalence of neurological conditions, urgent need for effective gene therapies, and ongoing research and development efforts targeting disorders such as Parkinson's disease and Alzheimer’s disease, and certain genetic neurodegenerative disorders.

By Adeno-Associated Virus Serotype

• AAV1
• AAV2
• AAV5
• AAV9

The AAV9 segment is expected to account for largest revenue share owing to efficient transduction with this serotype across various tissues and organs, including the central nervous system and the heart. Its broad tropism and ability to cross the blood-brain barrier make AAV9 a preferred serotype for addressing neurological disorders and cardiovascular diseases.

By End-User

• Hospitals
• Research Institutes
• Biopharmaceutical Companies

Among the end user segments, the biopharmaceutical companies segment is expected to account for majority revenue share. This projection is supported by increasing collaborations and partnerships between biopharmaceutical companies and research institutions for gene therapy development. Biopharmaceutical companies, with their resources and expertise, are well-positioned to drive advancements, conduct clinical trials, and bring AAV vector-based gene therapies to market.

By Therapeutic Area

• Rare Diseases
• Oncology
• Cardiovascular Diseases
• Genetic Disorders

The oncology segment is expected to account for largest revenue share over the forecast period, supported by increasing focus on developing gene therapies for various cancers, leveraging the targeted and sustained gene expression capabilities of AAV vectors. Ongoing clinical trials and research initiatives addressing oncological conditions contribute to the strong outlook for AAV vectors in the field of oncology.

By Region

North America

• United States
• Canada

Europe

• Germany
• United Kingdom
• France
• Italy
• Spain
• Russia
• Poland
• Benelux
• Nordic
• Rest of Europe

Asia Pacific

• China
• Japan
• India
• South Korea
• ASEAN
• Australia New Zealand
• Rest of Asia Pacific

Latin America

• Brazil
• Mexico
• Argentina

Middle East Africa

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

The global Adeno-Associated Virus (AAV) vector-based gene therapy market is divided into five key regions: North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa. North America market growth is driven majorly by revenue contribution from the United States, which has been a prominent player in the gene therapy sector. Factors such as robust research and development activities, supportive regulatory environment, and substantial investments contribute to the North America leading among the other regional markets. Europe market revenue growth is supported by substantial contribution from countries such as the United Kingdom and Germany, which also have been demonstrated significant growth in gene therapy research and development. Asia Pacific market growth is driven by rising investments in biotechnology and healthcare infrastructure in China and Japan.

Some common factors driving overall market growth include advancements in vector technologies, expanding applications across various therapeutic areas, and increased collaborations between biopharmaceutical companies and research institutions.

Leading Companies in Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market & Competitive Landscape:

The competitive landscape in the Adeno-Associated Virus (AAV) vector-based gene therapy market is dynamic, characterized by the presence of major players striving to maintain and enhance their positions. Leading companies are adopting strategic moves to stay competitive, with one key strategy being continuous investments in research and development to pioneer new AAV serotypes and improve vector engineering technologies. Collaborations and partnerships with research institutions and biopharmaceutical companies enable knowledge exchange and broaden the therapeutic applications of AAV vectors.

Also, strategic acquisitions contribute to market consolidation and expansion of service offerings. Companies are also focusing on optimizing vector production methods to address manufacturing challenges and meet the increasing demand for AAV vectors. This competitive landscape highlights commitment to innovation and collaboration as key drivers for success in the evolving gene therapy market.

These companies include:

• BioMarin Pharmaceutical
• Roche (Spark Therapeutics)
• Sangamo
• Pfizer
• Sarepta Therapeutics
• Freeline Therapeutics
• Regenxbio
• Amicus Therapeutics
• NightstaRx Ltd.
• Solid Biosciences
• Voyager Therapeutics
• UniQure
• Abeona Therapeutics
• Aldevron (acquired by Danaher)
• Oxford BioMedica

Recent Development:

January 2024: Voyager Therapeutics, Inc. announced a strategic collaboration and capsid license agreement with Novartis Pharma AG, which is a subsidiary of Novartis AG, to advance gene therapies for Huntington’s Disease (HD) and Spinal Muscular Atrophy (SMA). Novartis will gain a target-exclusive license for Voyager’s TRACER capsids and related intellectual property for HD and SMA, with both companies collaborating on a preclinical gene therapy candidate for HD. Novartis assumes exclusive access and responsibility for development and commercialization of TRACER capsids for SMA, and obtains global rights to Voyager’s AAV gene therapy for HD, utilizing TRACER capsids, with Voyager handling preclinical development and Novartis overseeing clinical and commercialization efforts for the HD program.

July 2023: AstraZeneca reported that its subsidiary, Alexion, has entered into an agreement to acquire the early-stage rare disease gene therapy portfolio of US drugmaker Pfizer for up to US$1 billion, along with royalties on sales. This strategic move by the British drugmaker is aimed at bolstering its commitment to new genetic therapies. The acquisition includes a range of innovative AAV capsids, which are demonstrated to be effective in delivering therapeutic gene cargos for gene therapy and gene editing. The deal is expected to enhance both Alexion and AstraZeneca's capabilities in the field of genomic medicine, marking a significant step in advancing their presence in the evolving landscape of rare disease treatments.

July 2023: Sangamo Therapeutics, Inc. announced the signing of an evaluation and option agreement with Prevail Therapeutics, which is a wholly owned subsidiary of Eli Lilly and Company. Under this agreement, Prevail has obtained rights to assess specific proprietary AAV capsids developed by Sangamo. Prevail also holds the option to license these capsids for multiple undisclosed neurological targets. These AAV capsids, created through Sangamo's SIFTER (Selecting In vivo For Transduction and Expression of RNA) capsid engineering platform, have demonstrated potential for highly efficient delivery of investigatory gene therapy constructs to the central nervous system during pre-clinical studies.

Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Research Scope:

Report Metric	Report Details
Market size available for the years	2021-2023
Base Year	2023
Forecast Period	2024-2032
Compound Annual Growth Rate (CAGR)	15.1%
Segment covered	Gene Therapy Application, AAV Serotype, End-User, Therapeutic Area
Regions Covered	North America: The U.S.Canada Latin America: Brazil, Mexico, Argentina, Rest of Latin America Asia Pacific: China, India, Japan, Australia New Zealand, ASEAN, Rest of Asia Pacific Europe: Germany, The U.K., France, Spain, Italy, Russia, Poland, BENELUX, NORDIC, Rest of Europe The Middle East Africa: Saudi Arabia, United Arab Emirates, South Africa, Egypt, Israel, and Rest of MEA
Fastest Growing Country in Europe	UK
Largest Market	North America
Key Players	BioMarin Pharmaceutical, Roche (Spark Therapeutics), Sangamo, Pfizer, Sarepta Therapeutics, Freeline Therapeutics, Regenxbio, Amicus Therapeutics, NightstaRx Ltd., Solid Biosciences, Voyager Therapeutics, UniQure, Abeona Therapeutics, Aldevron (acquired by Danaher), Oxford BioMedica