AAV Vector market

AAV Based Gene Therapy, AAV Manufacturing and AAV Technology Market Overview

The global AAV vector market (AAV based gene therapy and AAV manufacturing) is projected to grow from USD 1.9 billion in 2022 to USD 11.1 billion by 2035, exhibiting a CAGR of 14% during the forecast period. Given the potential of gene therapies in targeting the underlying cause of a disease at cellular level,  the demand for such therapies has increased considerably over the past few years. Currently, more than 285 gene therapies are being evaluated in different phases of clinical development.  Further, various gene therapy developers have raised more than USD 10 billion capital during the year 2021.  With the growing interest in such therapies, the demand for novel delivery vectors has also increased. Among various gene delivery vectors available, adeno-associated viral vectors (AAV) have emerged as one of the most efficient viral vectors.  Till now, there are six approved AAV based gene therapy, with five of them having received US FDA approval, the latest approval coming in June 2023. A number of adeno associated viral vector based therapies are also being evaluated in different clinical trials. In fact, it has been observed that clinical trials evaluating AAV based gene therapy have increased at a growth rate of ~30% over the last few years.  Further, it is worth mentioning that over 50 clinical trials are expected to complete in the next three years.  In order to cater to the demand, close to 100 companies, across the globe, have emerged for the development of AAV based gene therapy and AAV manufacturing. In fact, a number of these companies also offer advanced AAV technology platforms, enabling the processing of adeno-associated viral vectors and related therapies across different scales of operation from small scale to large scale. Looking at the adoption and acceptance of AAV vector as a safe delivery option for gene therapy, the market for AAV based gene therapy developers is likely to witness healthy market growth during the forecast period. This market growth will be driven by the support provided by AAV manufacturing companies and AAV technology providers.

Key Market Insights

The Adeno-Associated Viral Vectors / AAV Vector Market: Focus on AAV Based Gene Therapy and AAV Manufacturing by Type of Therapy (Gene Augmentation, Immunotherapy and Others), Type of Gene Delivery Method Used (Ex vivo and In vivo), Target Therapeutic Area (Genetic Disorders, Haematological Disorders, Infectious Diseases, Metabolic Disorders, Muscle Disorders, Ophthalmic Disorders, Neurological Disorders and Others), Application Area (Gene Therapy, Cell Therapy and Vaccine), Scale of Operation (Preclinical, Clinical and Commercial) and Geographical Regions (North America, Europe, Asia Pacific, MENA, Latin America and Rest of the World): Industry Trends and Global Forecasts, 2022-2035 market report features an extensive study on the current market landscape, market size and future opportunities available to the AAV based gene therapy developers, AAV manufacturing companies and AAV technology providers. The market report offers an informed opinion on the likely evolution and underlines an in-depth analysis, highlighting the key drivers and the market trends related to this evolving segment of the pharmaceutical industry. Key takeaways of the AAV vector market are briefly discussed below.

AAV Based Gene Therapy Market Landscape: Elevidys Becomes the Latest AAV Gene Therapy to Receive Approval

Novartis Gene Therapies was the first company to receive EMA approval for an AAV based gene therapy in 2012, when Glybera received marketing authorization for treatment of inherited metabolic disorder lipoprotein lipase (LPL) deficiency (LPLD). The journey for the drug was short as it never received a go ahead from the FDA and was withdrawn from European market in 2017. Although the event questioned the development of AAV as a delivery vector for gene therapies, the pharmaceutical industry has seen six successful approvals since 2019. Zolgensma (Novartis Gene Therapies), Luxturna (Spark Therapeutics), Hemgenix (CSL Behring / UniQure), Roctavian (BioMarin Pharmaceuticals), Upstaza (PTC Therapeutics) and recently approved Elevidys (Sarepta Therapeutics) are six AAV based gene therapy available in either the US or EU markets for treatment of various indications. Zolgensma uses recombinant AAV9 vector for delivery of SMN1 gene to treat patients with spinal muscular atrophy. According to the financial information provided by Novartis Gene Therapies, Zolgensma generated net sales of USD 1,370 million in full year 2022 and is amongst the top 10 products in the company’s innovative medicines division. Presently, more than 550 AAV based gene therapy are being evaluated by drug developers worldwide, details of which have been captured in our market report.

Clinical Trials Landscape: North America is the Hub for Clinical Trials Evaluating AAV Based Gene Therapy 

Over the past years, more than 155 clinical trials evaluating AAV gene therapy have been registered across different regions of the world. We have identified more than 120 active clinical trials worldwide. More than 7,000 patients are enrolled in these clinical trials, geographical distribution for which is available in this market report. In November 2022, MeiraGTx started recruiting patients in a phase I clinical trial of its AAV gene therapy AAV-GAD for Parkinson’s disease. Parkinson’s disease is an indication in early stages of evaluation for treatment with AAV based gene therapy. In March 2023, Asklepios BioPharmaceutical, a subsidiary of Bayer AG, announced the presentation of clinical trial results from a phase 1b study of its AAV gene therapy AB-1005 at AD/PD™ 2023 Advances in Science & Therapy Conference in Sweden. The candidate is also being developed for the treatment of mild to moderate Parkinson’s disease. The evaluation of AAV based gene therapy for treatment of neurological disorders is expected to be a market driver in the long term. 

AAV Manufacturing Companies: A Number of Manufacturers Have Developed Their Proprietary AAV Technology to Support Manufacturing

More than 80 companies across the globe have the required capabilities to manufacture adeno-associated viral vectors. At present, majority of the companies have preclinical and clinical batch manufacturing capabilities. Of the identified AAV manufacturing companies 43% also have capability to cater to large scale commercial manufacturing demand. Companies like Thermo Fisher Scientific are also developing AAV manufacturing technologies that can help the drug developers to scale production from small research scale to large scale commercial production. Catering to the growing demand for AAV vectors, Thermo Fisher Scientific launched Gibco AAV-MAX Helper-Free AAV Production System in November 2022. Another company, Charles River Laboratories launched a nAAVigation platform to support AAV manufacturing in October 2022. Charles River Laboratories platform claims to reduce the time from process development to large scale GMP manufacturing by 55%. Many more AAV manufacturing CMO and CDMO are developing AAV technology platforms for efficient manufacturing. Such technological developments will support market growth during the forecast period.

AAV Market Trends: Partnerships, Collaborations, Patents and More

Multiple partnerships and collaborations have been established between the stakeholders in order to enhance development activity in this growing segment of the pharmaceutical industry. For AAV based gene therapy developers, product development and commercialization agreements are the most popular engagement model. However, as the AAV gene therapies move from preclinical to clinical and commercial stages of development, there has been an increase in the demand for service of AAV manufacturing companies. Such companies are engaging in partnerships and collaborations, not only with the AAV based gene therapy developers but also with other manufacturers to enhance their service portfolio. In May 2023, Forge Biologics collaborated with LabCorp to provide access to good manufacturing practice (GMP) scale manufacturing capabilities, drug development services and scientific expertise to their clients in the AAV market.

The growing interest in AAV vector market is also evident from the patents filed for protection of intellectual property related to AAV technology. Close to 4,400 patents related to adeno-associated viral vectors have been analyzed in our market report. On the basis of patents analysis, Baxalta, Biomarin, Genzyme, Regenxbio, Spark Therapeutics and Voyager Therapeutics are the most popular AAV based gene therapy drug developers from the pharmaceutical industry. Considering the prevalent trends and projected opportunity associated with the overall adeno-associated viral vectors industry, we believe that the AAV vector market is anticipated to grow at a health CAGR during the forecast period.

AAV Vector Market Size: North America Holds the Largest Market Share of the AAV Based Gene Therapy Market

The global AAV vectors market was estimated to be USD 1.9 billion in 2022. In 2023 alone, there have been three key approvals. Another drug, Hemgenix from CSL Behring, which received FDA approval for treatment of hemophilia B in November 2022, witnessed its first sale in June 2023. Driven by the launch of new AAV based gene therapy candidates and a strong late stage pipeline, the AAV vectors market size is estimated to reach USD 11.1 billion by 2035, exhibiting a CAGR of 14% during forecast period. North America currently holds the largest market share, essentially owing to the approval of majority AAV based gene therapy candidates in the US. Additionally, the North American market has also witnessed adoption of this gene therapy segment by the patients. We expect the market in North America to continue to hold the largest market share during the forecast period of the report.

Leading AAV Based Gene Therapy Developers and AAV Manufacturing Companies

Examples of key players engaged in adeno-associated viral vectors industry / AAV vector market (which have also been profiled in this report) include (in alphabetical order), Abeona Therapeutics, Aldevron (acquired by Danaher), Oxford BioMedica, Sanofi (CEPiA, Sanofi Pasteur, Genzyme), WuXi AppTec, and YPOSKESI. This market report includes an easily searchable, excel database of all AAV based gene therapy developers, AAV manufacturing companies and AAV technology providers worldwide.

Recent Developments in AAV Vector Market

Several recent developments have taken place in the field of adeno-associated viral vectors. We’ve outlined some of these recent initiatives below. These developments, even if they took place post the release of our market report, substantiate the overall market trends that we’ve outlined in our analysis.

• In July 2023, Regenxbio announced interim clinical trial results from two phase II clinical trials evaluating ABBV-RGX-314 for treatment of ophthalmological and genetic disorders. The AAV based gene therapy is being developed in collaboration with AbbVie.
• In April 2023, GenSight Biologics withdrew its MAA application with EMA for its drug LUMEVOQ, which was under late stage development for treatment of LHON.

Scope of the Report

The market research report presents an in-depth analysis of the various AAV based gene therapy developers, AAV manufacturing companies and AAV technology providers engaged in the AAV vector market, across different segments, as defined in the table below:

The market report features an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this industry. Amongst other elements, the market research report includes:

• An executive summary of the key insights captured during our research. It offers a high-level view on the current state of the adeno-associated viral vectors market and its likely evolution in the mid to long term.
• A general overview of the various types of viral and non-viral vectors. It includes a detailed discussion on structure and design, life cycle and applications of adeno-associated viral vectors. The chapter concludes with a discussion on the various advantages and challenges related to adeno-associated viral vectors.
• A detailed overview of the overall market landscape of  AAV based gene therapy, including information on their phase of development (marketed, clinical, preclinical and discovery), key therapeutic areas (genetic disorders, haematological disorders, infectious diseases, metabolic disorders, muscle disorders, neurological disorders, ophthalmic disorders and others), type of gene / molecule targeted, type of therapy (gene augmentation, immunotherapy and others), type of gene delivery method used (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any).
• An overview of the current status of the market with respect to the players engaged in the AAV manufacturing, featuring information on the year of establishment, company size, location of headquarters, type of product manufactured (vector and gene therapy / cell therapy / vaccine), location of manufacturing facilities, type of manufacturer (in-house and contract services), scale of operation (preclinical, clinical and commercial) and application area (gene therapy, cell therapy, vaccine and others).
• An overview of the technologies offered / developed by the companies engaged in the AAV market, including a detailed analysis based on the type of AAV technology (viral vector related platform, non-viral vector related platform and others), scale of operation (preclinical, clinical and commercial), application area (gene therapy, cell therapy, vcaccine and others) and most prominent players within this market segment, in terms of number of technologies.
• Detailed profiles of marketed and late stage (phase II / III and above) AAV based gene therapy, along with information on the development timeline of the therapy, current development status, mechanism of action, affiliated AAV technology, patent portfolio, dosage and manufacturing details, as well as details related to the developer company.
• Tabulated profiles of AAV manufacturing companies (shortlisted based on proprietary criterion). Each profile features an overview of the company / organization, its financial performance (if available), vector manufacturing related capabilities and an informed future outlook.
• A region-wise, company competitiveness analysis, highlighting key players engaged in AAV manufacturing, based across key geographical areas, featuring a four-dimensional bubble representation, which takes into consideration supplier strength (based on experience in this field), manufacturing strength (type of product manufactured, number of manufacturing facilites and number of application areas), service strength (scale of operation, and geographical reach) and company size (small, mid-sized and large).
• A detailed competitiveness analysis of adeno-associated viral vector technology platforms, taking into consideration the supplier strength (based on the year of establishment of developer) and key AAV technology specifications, such as purpose of technology, scale of operation and application area(s).
• An in-depth analysis of completed, ongoing and planned clinical trials, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.
• An analysis of recent collaborations and partnership agreements inked in this market since 2017; it includes details of deals that were / are focused on AAV manufacturing, which were analyzed on the basis of year of partnership, type of partnership (product commercialization agreement, product / technology licensing agreement, product development agreement, product manufacturing agreement, merger / acqusition, research and development agreement, process development  / optimization agreement, service alliance, supply agreement, clinical trial agreement and others), therapeutic area (neurological disorders, ophthalmic disorders, genetic disorders, musculoskeletal disorders, cardiovascular disorders, hematological disorders, infectious diseases, metabolic disorders and others), type of partner (industry and non-industry) and regional distribution of partnerships. 
• An insightful analysis of the companies that have likelihood of establishing partnerships with adeno associated viral vector and gene therapy product manufacturers, based on several parameters, such as developer strength, product strength, therapeutic area and pipeline strength (preclinical and clinical ).
• An in-depth analysis of various patents that have been filed / granted for adeno associated viral vector based therapies since 2017, based on several relevant parameters, such as type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation.
• An analysis of the various start-ups engaged in the development of AAV based gene therapy, based on relevant parameters, such as number of candidates in discovery, preclinical and clinical phases of development, number of patents and number of partnerships established.
• An insightful analysis, highlighting the various factors that need to be taken into consideration by AAV based gene therapy developers to facilitate decision making to manufacture their respective products in-house or engage the services of a CMO. Further, the analysis highlights all the key parameters that must be considered by players based on company sizes (small, mid-sized, and large), while taking the aforementioned decision.

One of the key objectives of this market report was to evaluate the current market size and the future opportunity associated with the adeno-associated viral vectors / AAV vector market, over the forecast period. Based on various parameters, such as the likely increase in number of clinical trials, anticipated growth in target patient population, existing price variations across different types of vectors, and the anticipated success of gene therapy products (considering both approved and late-stage clinical candidates), we have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2022-2035. In order to provide a detailed future outlook, our projections have been segmented on the basis of type of therapy (gene augmentation, immunotherapy and others), type of gene delivery method used (ex vivo and in vivo), target therapeutic area (genetic disorders, hematological disorders, infectious diseases, metabolic disorders, ophthalmic disorders, muscle disorders, neurological disorders and others), application area (gene therapy, cell therapy and vaccine), scale of operation (preclinical, clinical and commercial), and geographical region (North America, Europe, Asia Pacific, MENA, Latin America and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s market growth.

The opinions and insights presented in this study were also influenced by discussions held with senior stakeholders in the industry. The market report features detailed transcripts of interviews held with the following industry and non-industry players:

• Arun Srivastava (Professor of Genetics and Chief of Division of Cellular & Molecular Therapy, University of Florida)
• Abhishek Chatterjee (Professor, Boston College)
• Adam Lauber (Chief Financial Officer, Andelyn Biosciences)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this research report are in USD, unless otherwise specified.

Frequently Asked Questions

• What is an AAV gene therapy?
• Which AAV based gene therapy candidates are present in the current development pipeline? Which disease indications are targeted by such products?
• Who are the leading AAV manufacturing companies engaged in AAV production?
• Who are the leading AAV gene therapy companies?
• Which type of adeno-associated viral vector related technologies are presently offered / being developed by the players in this market?
• Which geographies are the most active in conducting clinical trials related to adeno-associated viral vectors?
• Which partnership models are commonly adopted by industry and non-industry stakeholders?
• Which companies are likely to partner with adeno associated viral vector and gene therapy product manufacturers?
• What are the different initiatives undertaken by start-ups for the development of adeno-associated viral vectors in the recent past?
• Which factors are likely to influence the decision of manufacturing the adeno-associated viral vector in-house or via outsourcing?
• How big is the AAV based gene therapy market?
• How big is the AAV manufacturing market size?
• What is the anticipated AAV market growth during the forecast period?