Adeno-Associated Viral (AAV) Vector Market: Focus on Drugs, Manufacturers and Technologies, 2022-2035

Adeno-Associated Viral (AAV) Vector Market: Focus on Drugs, Manufacturers and Technologies, 2022-2035

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 (AAV) vectors have emerged as one of the most efficient viral vectors. Till now, the USFDA has approved two adeno-associated viral vectors based drugs, LUXTURNA® and ZOLGENSMA®. 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 adeno-associated viral vector based therapies have increased at a growth rate of ~30% over the last few years. Further, it is worth mentioning that over 50 trials are expected to complete in the next three years.

In order to cater to the demand, close to 100 players, across the globe, have emerged for the development and manufacturing of adeno-associated viral vectors. In fact, a number of these companies also offer advanced technology platforms, enabling the processing of adeno-associated viral vectors and related therapies across different scales of operation. Various industry and non-industry players are actively engaged in research and development of novel gene delivery technologies, which are safe and effective. This is evident from the patents filed for protection of intellectual property related to such technologies. Additionally, multiple partnerships and collaborations have been established between the stakeholders in order to enhance development activity in this industry. Considering the prevalent trends and projected opportunity associated with the overall adeno-associated viral vector domain, we believe that the market is anticipated to witness substantial growth in the foreseen future.

The “Adeno-Associated Viral (AAV) Vector Market: Focus on Drugs, Manufacturers and Technologies 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” report features an extensive study on the current market landscape, offering an informed opinion on the likely evolution and future opportunities associated with the adeno-associated viral vector market. The study underlines an in-depth analysis, highlighting the key drivers and the market trends related to this evolving segment of the pharmaceutical industry. Amongst other elements, the 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 therapeutic vaccines 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 adeno-associated viral vector based therapies, 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 manufacturing of adeno-associated viral vectors, 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 enagaged in this domain, including a detailed analysis based on the type of 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 domain, in terms of number of technologies.

Detailed profiles of marketed and late stage (phase II / III and above) adeno-associated viral vector based therapies, along with information on the development timeline of the therapy, current development status, mechanism of action, affiliated technology, patent portfolio, dosage and manufacturing details, as well as details related to the developer company.

Tabulated profiles of players having capability to manufacture adeno-associated viral vectors (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 the manufacturing of adeno-associated viral vector, 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 platforms, taking into consideration the supplier strength (based on the year of establishment of developer) and key technology specifications, such as purpose of technology, scale of operation and application area(s).

An in-depth analysis of completed, ongoing and planned clinical studies, 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 domain since 2017; it includes details of deals that were / are focused on the manufacturing of vectors, 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 adeno-associated viral vectors-based therapies, 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 adeno-associated viral vector manufacturers 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 report was to evaluate the current market size and the future opportunity associated with the adeno-associated viral vector market, over the coming decade. Based on various parameters, such as the likely increase in number of clinical studies, 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 growth.

The opinions and insights presented in this study were also influenced by discussions held with senior stakeholders in the industry. The 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 report are in USD, unless otherwise specified.

MARKET SEGMENTATIONS

Adeno-Associated Viral (AAV) Vector Market – Focus on Drugs, Manufacturers and Technologies: Market Segmentations

Market Segments Details

Forecast Period 2022 – 2035

Type of Therapy Gene Augmentation

Immunotherapy

Others

Type of Gene Delivery Method Used Ex vivo

In vivo

Target Therapeutic Area Genetic Disorders

Hematological Disorders

Infectious Diseases

Metabolic Disorders

Ophthalmic Disorders

Muscle Disorders

Neurological Disorders

Others

Scale of Operation Preclinical

Clinical

Commercial

Application Area Gene Therapy

Cell Therapy

Vaccines

Geographical Regions North America

Europe

Asia-Pacific

MENA

Latin America

Rest of the World

Source: Roots Analysis

KEY QUESTIONS ANSWERED

Which adeno-associated viral vector-based therapy candidates are present in the current development pipeline? Which disease indications are targeted by such products?

Who are the leading players (contract service providers and in-house manufacturers) engaged in the development of adeno-associated viral vectors?

Which type of adeno-associated viral vector related technologies are presently offered / being developed by the players in this domain?

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 is the current and future market opportunity likely to be distributed across key market segments?

RESEARCH METHODOLOGY

The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market may evolve across different regions and technology segments. Wherever possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include:

Annual reports

Investor presentations

SEC filings

Industry databases

News releases from company websites

Government policy documents

Industry analysts’ views

While the focus has been on forecasting the market over the period 2022-2035, the report also provides our independent view on various technological and non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.

CHAPTER OUTLINES

Chapter 2 is an executive summary of the key insights captured during our research. It offers a high-level view on the current state of the therapeutic vaccines market and its likely evolution in the mid to long term.

Chapter 3 is 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.

Chapter 4 includes information on over 550 therapies related to adeno-associated viral vectors that are currently approved or are in different stages of development. It features a detailed analysis of the therapies, based on several relevant parameters, such as key therapeutic areas (genetic disorders, hematological disorders, immunological disorders, infectious diseases, metabolic disorders, muscle disorders, neurological disorders, ophthalmic disorders and others), phase of development (marketed, clinical, preclinical and discovery), type of gene / molecule targeted, type of gene delivery method used (ex vivo and in vivo), type of therapy (gene augmentation, immunotherapy and others), route of administration and special drug designation (if any).

Chapter 5 provides a detailed overview of close to 80 companies, featuring both contract service providers and in-house manufacturers that are actively involved in the manufacturing of adeno-associated viral vectors. The chapter provides details 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).

Chapter 6 features an in-depth analysis of adeno-associated viral vector based technologies offered / developed by the players in this domain, based on the type of technology (viral vector and non-viral vector related platform), scale of operation (preclinical, clinical and commerical), application area (gene therapy, cell therapy, vaccine and others) and leading technology providers.

Chapter 7 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the company’s patent portfolio and collaborations focused on its gene therapy product / technology. Further, the chapter highlights the detailed profiles of drugs that are in advanced stages of clinical development (phase II / III and above). Each drug profile provides information on the development timeline of the therapy, current developmental status, route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, affiliated technology, patent portfolio strength, clinical trials and collaborations (if any).

Chapter 8 features detailed profiles of players having capability to manufacture adeno-associated viral vectors based in North America, Europe and Asia-Pacific & RoW. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities and an informed future outlook.

Chapter 9 presents a detailed competitiveness analysis of adeno-associated viral vector manufacturers across key geographical areas, featuring a four-dimensional bubble representation, taking into consideration supplier strength (based on its experience in this field), manufacturing strength (type of product manufactured, number of manufacturing facilities and number of application area), service strength (scale of operation and geographical reach) and company size (small, mid-sized and large).

Chapter 10 provides an insightful competitiveness analysis of adeno-associated viral vector related platforms. It highlights the leading players in this domain, taking into consideration the supplier strength (based on the year of establishment) and key technology specifications, such as purpose of technology, scale of operation and application area(s).

Chapter 11 provides information on more than 155 completed, ongoing and planned clinical trials of adeno-associated viral vectors, based on different parameters, such as trial registration year, trial phase, trial recruitment status, study design, target disease indication(s), type of sponsor / collaborator, leading industry sponsors / collaborators (in terms of number of trials conducted), enrolled patients population and regional distribution.

Chapter 12 provides a detailed analysis of the recent partnerships established since 2017. It includes analysis based on relevant parameters, such as year of agreement, type of agreement (product commercialization agreement, product / technology licensing agreement, product development agreement, product manufacturing agreement, merger / acquisition, 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.

Chapter 13 highlights potential strategic partners for 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). The analysis aims to provide the necessary inputs to the product developers, enabling them to make the right decisions to collaborate with industry stakeholders with relatively more initiatives in the domain.

Chapter 14 provides an overview of the various patents that have been filed / granted for therapies related to adeno-associated viral vectors, since 2017, based on several relevant parameters, such as type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed / granted). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation.

Chapter 15 features an analysis of the various start-ups engaged in manufacturing of adeno-associated viral vector-based therapies, based on relevant parameters, such as number of candidates in discovery, preclinical and clinical phases of development, number of partnerships, number of patents and number of deals inked.

Chapter 16 presents an insightful analysis that highlights the various factors that need to be taken into consideration by adeno-associated viral vector manufacturers to facilitate decision making to manufacture their respective therapeutics in-house or engage the services of a CMOs. Further, the analysis highlights all the key parameters that must be considered by players of different company sizes (small, mid-sized, and large) while taking the aforementioned decision.

Chapter 17 presents a comprehensive market forecast analysis, highlighting the likely growth of adeno-associated viral vector market till the year 2035. We have segmented the financial opportunity 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), scale of operation (preclinical, clinical and commercial), application area (gene therapy, cell therapy and vaccine) and geography (North America, Europe, Asia Pacific, MENA, Latin America and rest of the world). Due to the uncertain nature of the market, we have presented three different growth tracks outlined as the conservative, base and optimistic scenarios.

Chapter 18 is a summary of the overall report, which presents insights on the contemporary market trends and the likely evolution of the adeno-associated viral vector market.

Chapter 19 is a collection of transcripts of the interviews conducted with representatives from renowned organizations that are engaged in the adeno-associated viral vector domain. In this study, we spoke to Arun Srivastava (Professor of Genetics and Chief of Division of Cellular & Molecular Therapy, University of Florida), Abhishek Chatterjee (Professor, Boston College) and Adam Lauber (Chief Financial Officer, Andelyn Biosciences).

Chapter 20 is an appendix that contains tabulated data and numbers for all the figures in the report.

Chapter 21 is an appendix that provides the list of companies and organizations mentioned in the report.