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AAV Vector Market: Focus on Drugs, Manufacturers and Technologies by Type of Therapy, Type of Gene Delivery Method Used, Target Therapeutic Area, Application Area, Scale of Operation and Geographical Regions: Industry Trends and Global Forecasts, 2022-2035

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    Report

  • 250 Pages
  • August 2022
  • Region: Global
  • Roots Analysis
  • ID: 5659470
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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 ~30% growth rate 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 / AAV vector domain, we believe that the market is anticipated to witness substantial growth in the foreseen future.

Scope of the Report

The “Adeno-Associated Viral Vector / 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.

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?

Table of Contents

1. PREFACE
1.1. Scope of the Report
1.2. Market Segmentation
1.3. Research Methodology
1.4. Key Questions Answered
1.5. Chapter Outlines

2. EXECUTIVE SUMMARY
3. INTRODUCTION
3.1. Chapter Overview
3.2. Viral and Non-Viral Vectors
3.2.1. Viral Vectors
3.2.1.1. Adenovirus Vectors
3.2.1.2. Adeno-Associated Viral Vectors
3.2.1.3. Lentivirus Vectors
3.2.1.4. Retrovirus Vectors
3.2.1.5. Other Viral Vectors
3.2.1.5.1. Alphavirus
3.2.1.5.2. Foamy Virus
3.2.1.5.3. Simian Virus
3.2.1.5.4. Vaccinia Virus
3.2.1.5.5. Chimeric Viral Vectors
3.2.1.5.6. Herpes Simplex Virus
3.2.1.5.7. Sendai Virus
3.2.2. Non-Viral Vectors
3.2.2.1. Plasmid DNA
3.2.2.2. Oligonucleotides
3.2.2.3. Liposomes, Lipoplexes, and Polyplexes
3.2.2.4. Other Non-Viral Vectors
3.3. Adeno-Associated Viral Vectors
3.3.1. Structure and Design
3.3.2. Adeno-Associated Viral Vector Life Cycle
3.3.3. Applications of Adeno-Associated Viral Vectors
3.3.3.1. Gene Therapy
3.3.3.2. Vaccination
3.3.4. Advantages of Adeno-Associated Viral Vectors
3.3.5. Challenges Related to Adeno-Associated Viral Vectors
3.4. Concluding Remarks

4. ADENO-ASSOCIATED VIRAL VECTOR BASED THERAPY: MARKET LANDSCAPE
4.1. Chapter Overview
4.2. Adeno-Associated Viral Vector Based Therapy: Overall Market Landscape
4.2.1. Analysis by Phase of Development
4.2.2. Analysis by Therapeutic Area(s)
4.2.3. Analysis by Type of Gene / Molecule Targeted
4.2.4. Analysis by Type of Therapy
4.2.5. Analysis by Type of Gene Delivery Method Used
4.2.6. Analysis by Route of Administration
4.3. Adeno-Associated Viral Vector Based Therapy Candidates: Special Designations
4.3.1. Analysis by Special Designation(s) Awarded
4.4. Adeno-Associated Viral Vector Based Therapy: List of Developers
4.4.1. Analysis by Year of Establishment
4.4.2. Analysis by Company Size
4.4.3. Analysis by Location of Headquarters
4.4.4. Adeno-Associated Viral Vector Based Therapy: Leading Developers

5. ADENO-ASSOCIATED VIRAL VECTOR MANUFACTURERS: MARKET LANDSCAPE
5.1. Chapter Overview
5.2. Adeno-Associated Viral Vector Manufacturers: Overall Market Landscape
5.2.1. Analysis by Year of Establishment
5.2.2. Analysis by Company Size
5.2.3. Analysis by Location of Headquarters
5.2.4. Analysis by Type of Product(s) Manufactured
5.2.5. Analysis by Location of Vector Manufacturing Facilities
5.2.6. Analysis by Type of Manufacturer and Company Size
5.2.7. Analysis by Scale of Operation
5.2.8. Analysis by Location of Headquarters and Scale of Operation
5.2.9. Analysis by Application Area(s)

6. ADENO-ASSOCIATED VIRAL VECTOR MANUFACTURING TECHNOLOGIES: MARKET LANDSCAPE
6.1. Chapter Overview
6.2. Adeno-Associated Viral Vector Manufacturing Technologies
6.2.1. Analysis by Type of Technology
6.2.2. Analysis by Scale of Operation
6.2.3. Analysis by Application Area(s)
6.2.4. Analysis by Therapeutic Area
6.3. Adeno-Associated Viral Vector Technology: List of Developers
6.3.1. Analysis by Year of Establishment
6.3.2. Analysis by Company Size
6.3.3. Analysis by Location of Headquarters
6.3.4. Analysis by Company Size and Location of Headquarters
6.3.5. Adeno-Associated Viral Vector Technology: Leading Developers

7. DRUG PROFILES
7.1. Chapter Overview
7.2. Marketed Gene Therapies
7.2.1. LUXUTRNA® (Spark Therapeutics)
7.2.1.1. Company Overview
7.2.1.2. Development Timeline
7.2.1.3. Mechanism of Action
7.2.1.4. Target Indication(s)
7.2.1.5. Current Status of Development
7.2.1.6. Manufacturing, Dosage and Sales
7.2.2. ZOLGENSMA® (Novartis)
7.2.2.1. Company Overview
7.2.2.2. Development Timeline
7.2.2.3. Mechanism of Action
7.2.2.4. Target Indication(s)
7.2.2.5. Current Status of Development
7.2.2.6. Manufacturing, Dosage and Sales
7.3. Late Stage (Phase II/III and Above) Gene Therapies
7.3.1. AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.2. LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.3. NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.4. RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.5. AMT-061: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.6. OAV-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.7. PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.8. PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.9. PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.10. SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.11. SRP-9001: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.12. BMN 270: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.13. Lumevoq® (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
7.3.14. PTC-AADC: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results

8. COMPANY PROFILES
8.1. Chapter Overview
8.2. Abeona Therapeutics
8.2.1. Company Overview
8.2.2. Financial Information
8.2.3. AAV Vector Manufacturing related Capabilities
8.2.4. Recent Developments and Future Outlook
8.3 Aldevron (Acquired by Danaher)
8.3.1. Company Overview
8.3.2. Financial Information
8.3.3. AAV Vector Manufacturing related Capabilities
8.3.4. Recent Developments and Future Outlook
8.4 Oxford BioMedica
8.4.1. Company Overview
8.4.2. Financial Information
8.4.3. AAV Vector Manufacturing related Capabilities
8.4.4. Recent Developments and Future Outlook
8.5 Sanofi (CEPiA, Sanofi Pasteur, Genzyme)
8.5.1. Company Overview
8.5.2. Financial Information
8.5.3. AAV Vector Manufacturing related Capabilities
8.5.4. Recent Developments and Future Outlook
8.6. WuXi AppTec
8.6.1. Company Overview
8.6.2. Financial Information
8.6.3. AAV Vector Manufacturing related Capabilities
8.6.4. Recent Developments and Future Outlook
8.7. YPOSKESI
8.7.1. Company Overview
8.7.2. AAV Vector Manufacturing related Capabilities
8.7.3. Recent Developments and Future Outlook

9. COMPANY COMPETITIVENESS ANALYSIS
9.1. Chapter Overview
9.2. Methodology and Key Parameters
9.3. Adeno-Associated Viral Vector Manufacturers: In-house Manufacturers
9.3.1. Players based in North America
9.3.2. Players based in Europe
9.4. Adeno-Associated Viral Vector Manufacturers: Contract Manufacturing Organizations
9.4.1. Players based in North America
9.4.2. Players based in Europe
9.4.3. Players based in Asia-Pacific
9.5. Adeno-Associated Viral Vector Manufacturers: Both In-House and Contract Manufacturing Organizations
9.5.1. Players based in North America
9.5.2. Players based in Europe
9.5.3. Players based in Asia-Pacific

10. TECHNOLOGY COMPETITIVENESS ANALYSIS
10.1. Chapter Overview
10.2. Methodology and Key Parameters
10.3. Adeno-Associated Viral Vector Technology Platforms
10.3.1. Adeno-Associated Viral Vector Technology Platforms Developed by Companies
10.3.2. Adeno-Associated Viral Vector Technology Platforms Developed by Companies based in Europe and Asia-Pacific

11. CLINICAL TRIAL ANALYSIS
11.1. Chapter Overview
11.2. Scope and Methodology
11.3. Adeno-Associated Viral Vector Based Therapies: Clinical Trial Analysis
11.3.1. Analysis by Trial Registration Year
11.3.2. Analysis by Trial Registration Year and Enrolled Patient Population
11.3.3. Analysis by Trial Phase and Enrolled Patient Population
11.3.4. Analysis by Trial Status
11.3.5. Analysis by Study Design
11.3.6. Analysis by Therapeutic Area
11.3.7. Analysis by Target Disease Indication
11.3.8. Analysis by Type of Sponsor / Collaborator
11.3.9. Leading Industry Players: Analysis by Number of Registered Trials
11.3.10. Regional Analysis
11.3.10.1. Analysis by Number of Registered Trials
11.3.10.2. Analysis by Enrolled Patient Population

12. PARTNERSHIPS AND COLLABORATIONS
12.1. Chapter Overview
12.2. Partnership Models
12.3. Adeno-Associated Viral Vector based Therapies: List of Partnerships and Collaborations
12.3.1. Analysis by Year of Partnership
12.3.2. Analysis by Type of Partnership
12.3.3. Analysis by Year of Partnership and Type of Partner
12.3.4. Analysis by Type of Partnership and Type of Partner
12.3.5. Analysis by Therapeutic Area
12.3.6. Most Active Players: Analysis by Number of Partnerships
12.3.7. Regional Analysis
12.3.7.1. Intercontinental and Intracontinental Agreements
12.4. Adeno-Associated Viral Vector Manufacturers: List of Partnerships and Collaborations
12.4.1. Analysis by Year of Partnership
12.4.2. Analysis by Type of Partnership
12.4.3. Analysis by Year of Partnership and Type of Partner
12.4.4. Analysis by Type of Partnership and Type of Partner
12.4.5. Analysis by Therapeutic Area
12.4.6. Most Active Players: Analysis by Number of Partnerships
12.4.7. Regional Analysis
12.4.7.1. Intercontinental and Intracontinental Agreements

13. STRATEGIC PARTNER ANALYSIS
13.1. Chapter Overview
13.2. Adeno-Associated Viral Vector-based Therapy Manufacturers: Potential Strategic Partners
13.2.1. Methodology and Key Parameters
13.2.2. Adeno-Associated Viral Vector-based Therapy Manufacturers: Strategic Partner Analysis
13.2.2.1. Most Likely Partners
13.2.2.2. Likely Partners
13.2.2.3. Less Likely Partners
13.2.2.4. Least Likely Partners

14. PATENT ANALYSIS
14.1. Chapter Overview
14.2. Scope and Methodology
14.3. Adeno-Associated Viral Vectors: Patent Analysis
14.3.1. Analysis by Publication Year
14.3.2. Analysis by Annual Patent Application(s)
14.3.3. Analysis by Annual Granted Patents
14.3.4. Analysis by Geography
14.3.5. Analysis by CPC Symbols
14.3.6. Analysis by Type of Applicant
14.3.7. Leading Industry Players: Analysis by Number of Patents
14.4. Adeno-Associated Viral Vector Based Market: Patent Benchmarking
14.4.1. Analysis by Patent Characteristics
14.5. Adeno-Associated Viral Vectors: Patent Valuation
14.6. Leading Patents: Analysis by Number of Citations

15. START-UP HEALTH INDEXING
15.1. Chapter Overview
15.2. Start-ups Developing Adeno-Associated Viral Vectors
15.2.1. Analysis by Location of Headquarters
15.3. Benchmarking of Start-ups
15.3.1. Analysis by Pipeline Strength
15.3.2. Analysis by Pipeline Maturity
15.3.3. Analysis by Indication Diversity
15.3.4. Analysis by Number of Patents
15.3.5. Analysis by Partnership Activity
15.3.6. Start-ups Health Indexing

16. OUTSOURCING: GO / NO-GO FRAMEWORK
16.1. Chapter Overview
16.2. Outsourcing: Go / No-Go Framework
16.3. Adeno-Associated Viral Vector Developers Outsourcing: Go / No-Go Framework
16.3.1. Key Parameters and Assumptions
16.3.2. Methodology
16.3.3. Results and Interpretations
16.3.3.1. Small Companies
16.3.3.2. Mid-sized Companies
16.3.3.3. Large Companies

17. MARKET SIZING AND OPPORTUNITY ANALYSIS
17.1. Chapter Overview
17.2. Methodology and Key Assumptions
17.3. Adeno-Associated Viral Vector based Therapeutics Market, 2022-2035
17.3.1. Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Therapeutic Area
17.3.2. Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Type of Therapy
17.3.3. Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Type of Gene Delivery Method Used
17.3.4. Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Therapeutic Area
17.3.5. Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Route of Administration
17.3.6. Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Therapeutic Area
17.3.7. Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Geography
17.3.8. Adeno-Associated Viral Vector based Therapeutics Market, 2022-2035: Individual Product Sales Forecast
17.3.8.1. LUXTURNA
17.3.8.1.1. Target Patient Population
17.3.8.1.2. Sales Forecast
17.3.8.1.3. Net Present Value
17.3.8.1.4. Value Creation Analysis
17.3.8.2. ZOLGENSMA
17.3.8.2.1. Target Patient Population
17.3.8.2.2. Sales Forecast
17.3.8.2.3. Net Present Value
17.3.8.2.4. Value Creation Analysis
17.3.8.3. AGTC 501
17.3.8.3.1. Target Patient Population
17.3.8.3.2. Sales Forecast
17.3.8.3.3. Net Present Value
17.3.8.3.4. Value Creation Analysis
17.3.8.4. AMT-061
17.3.8.4.1. Target Patient Population
17.3.8.4.2. Sales Forecast
17.3.8.4.3. Net Present Value
17.3.8.4.4. Value Creation Analysis
17.3.8.5. BMN 270
17.3.8.5.1. Target Patient Population
17.3.8.5.2. Sales Forecast
17.3.8.5.3. Net Present Value
17.3.8.5.4. Value Creation Analysis
17.3.8.6. Lumevoq
17.3.8.6.1. Target Patient Population
17.3.8.6.2. Sales Forecast
17.3.8.6.3. Net Present Value
17.3.8.6.4. Value Creation Analysis
17.3.8.7. LYS-SAF302
17.3.8.7.1. Target Patient Population
17.3.8.7.2. Sales Forecast
17.3.8.7.3. Net Present Value
17.3.8.7.4. Value Creation Analysis
17.3.8.8. NFS-01
17.3.8.8.1. Target Patient Population
17.3.8.8.2. Sales Forecast
17.3.8.8.3. Net Present Value
17.3.8.8.4. Value Creation Analysis
17.3.8.9. OAV-101
17.3.8.9.1. Target Patient Population
17.3.8.9.2. Sales Forecast
17.3.8.9.3. Net Present Value
17.3.8.9.4. Value Creation Analysis
17.3.8.10. PF-06838435
17.3.8.10.1. Target Patient Population
17.3.8.10.2. Sales Forecast
17.3.8.10.3. Net Present Value
17.3.8.10.4. Value Creation Analysis
17.3.8.11. PF-06939926
17.3.8.11.1. Target Patient Population
17.3.8.11.2. Sales Forecast
17.3.8.11.3. Net Present Value
17.3.8.11.4. Value Creation Analysis
17.3.8.12. PF-07055480
17.3.8.12.1. Target Patient Population
17.3.8.12.2. Sales Forecast
17.3.8.12.3. Net Present Value
17.3.8.12.4. Value Creation Analysis
17.3.8.13. PTC-AADC
17.3.8.13.1. Target Patient Population
17.3.8.13.2. Sales Forecast
17.3.8.13.3. Net Present Value
17.3.8.13.4. Value Creation Analysis
17.3.8.14. RGX-314
17.3.8.14.1. Target Patient Population
17.3.8.14.2. Sales Forecast
17.3.8.14.3. Net Present Value
17.3.8.14.4. Value Creation Analysis
17.3.8.15. SPK-8011
17.3.8.15.1. Target Patient Population
17.3.8.15.2. Sales Forecast
17.3.8.15.3. Net Present Value
17.3.8.15.4. Value Creation Analysis
17.3.8.16. SRP-9001
17.3.8.16.1. Target Patient Population
17.3.8.16.2. Sales Forecast
17.3.8.16.3. Net Present Value
17.3.8.16.4. Value Creation Analysis
17.4. Adeno-Associated Viral Vector Manufacturers Market, 2022-2035
17.4.1. Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Phase of Development
17.4.2. Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Therapeutic Area
17.4.3. Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Application Area
17.4.4. Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Key Geographical Regions
17.5 Current and Future Market Opportunity for Commercial Products
17.5.1. Adeno-Associated Viral Vector Manufacturing Market for Commercial Products, 2022-2035: Distribution by Therapeutic Area
17.5.2. Adeno-Associated Viral Vector Manufacturing Market for Commercial Products, 2022-2035: Distribution by Application Area
17.5.3. Adeno-Associated Viral Vector Manufacturing Market for Commercial Products, 2022-2035: Distribution by Key Geographical Regions
17.6. Current and Future Market Opportunity for Clinical Candidates
17.6.1. Adeno-Associated Viral Vector Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Phase of Development
17.6.2. Adeno-Associated Viral Vector Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Application Area
17.6.3. Adeno-Associated Viral Vector Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Key Geographical Regions
17.7. Current and Future Market Opportunity for Preclinical Candidates
17.7.1. Adeno-Associated Viral Vector Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Type of Animal Model Used
17.7.2. Adeno-Associated Viral Vector Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Application Area
17.7.3. Adeno-Associated Viral Vector Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Key Geographical Regions

18. CONCLUDING REMARKS
19. EXECUTIVE INSIGHTS
19.1. Chapter Overview
19.2. Boston College
19.2.1. Organization Snapshot
19.2.2. Interview Transcript: Abhishek Chatterjee, Professor
19.3. University of Florida
19.3.1. Organization Snapshot
19.3.2. Interview Transcript: Arun Srivastava, Professor of Genetics and Chief of Division of Cellular & Molecular Therapy
19.4. Andelyn Biosciences
19.4.1. Organization Snapshot
19.4.2. Interview Transcript: Adam Lauber, Chief Financial Officer

20. APPENDIX 1: TABULATED DATA21. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
List Of Figures
Figure 3.1 Classification of Vectors
Figure 3.2 Structure of Adeno-Associated Viral Vector
Figure 4.1 Adeno-Associated Viral Vector based Therapies: Distribution by Phase of Development
Figure 4.2 Adeno-Associated Viral Vector based Therapies: Distribution by Therapeutic Area(s)
Figure 4.3 Adeno-Associated Viral Vector based Therapies: Distribution by Phase of Development and Therapeutic Area
Figure 4.4 Adeno-Associated Viral Vector based Therapies: Distribution by Type of Gene / Molecule Targeted
Figure 4.5 Adeno-Associated Viral Vector based Therapies: Distribution by Type of Therapy
Figure 4.6 Adeno-Associated Viral Vector based Therapies: Distribution by Type of Gene Delivery Method Used
Figure 4.7 Adeno-Associated Viral Vector based Therapies: Distribution by Route of Administration
Figure 4.8 Adeno-Associated Viral Vector based Therapies: Distribution by Special Designation(s) Awarded
Figure 4.9 Adeno-Associated Viral Vector based Therapy Developers: Distribution by Year of Establishment
Figure 4.10 Adeno-Associated Viral Vector based Therapy Developers: Distribution by Company Size
Figure 4.11 Adeno-Associated Viral Vector based Therapy Developers: Distribution by Location of Headquarters (Region-wise)
Figure 4.12 World Map Representation: Distribution by Geographical Location (Country-wise)
Figure 4.13 Leading Developers: Distribution by Number of Therapies in Pipeline
Figure 5.1 Adeno-Associated Viral Vector Manufacturers: Distribution by Year of Establishment
Figure 5.2 Adeno-Associated Viral Vector Manufacturers: Distribution by Company Size
Figure 5.3 Adeno-Associated Viral Vector Manufacturers: Distribution by Location of Headquarters (Region-wise)
Figure 5.4 Adeno-Associated Viral Vector Manufacturers: Distribution by Location of Headquarters (Country-wise)
Figure 5.5 Adeno-Associated Viral Vector Manufacturers: Distribution by Type of Product(s) Manufactured
Figure 5.6 Adeno-Associated Viral Vector Manufacturers: Distribution by Location of Vector Manufacturing Facilities (Region-wise)
Figure 5.7 Adeno-Associated Viral Vector Manufacturers: Distribution by Location of Vector Manufacturing Facilities (Country-wise)
Figure 5.8 Adeno-Associated Viral Vector Manufacturers: Distribution by Type of Manufacturer and Company Size
Figure 5.9 Adeno-Associated Viral Vector Manufacturers: Distribution by Scale of Operation
Figure 5.10 Adeno-Associated Viral Vector Manufacturers: Distribution by Application Area(s)
Figure 6.1 Adeno-Associated Viral Vector Manufacturing Technologies: Distribution by Type of Technology
Figure 6.2 Adeno-Associated Viral Vector Manufacturing Technologies: Distribution by Scale of Operation
Figure 6.3 Adeno-Associated Viral Vector Manufacturing Technologies: Distribution by Application Area(s)
Figure 6.4 Adeno-Associated Viral Vector Manufacturing Technologies: Distribution by Therapeutic Area(s)
Figure 6.5 Adeno-Associated Viral Vector Technology Developers: Distribution by Year of Establishment
Figure 6.6 Adeno-Associated Viral Vector Technology Developers: Distribution by Company Size
Figure 6.7 Adeno-Associated Viral Vector Technology Developers: Distribution by Location of Headquarters (Region-wise)
Figure 6.8 Adeno-Associated Viral Vector Technology: Distribution by Location of Headquarters (Country-wise)
Figure 6.9 Adeno-Associated Viral Vector Technology Developers: Distribution by Company Size and Location of Headquarters
Figure 6.10 Leading Developers: Distribution by Number of Technologies
Figure 7.1 Luxturna®: Development Timeline
Figure 7.2 Zolgensma®: Development Timeline
Figure 8.1 Abeona Therapeutics:Annual Revenues, 2016-Q1 2022 (USD Million)
Figure 8.2 Danaher: Annual Revenues, 2014-Q1 2022 (USD Billion)
Figure 8.3 Oxford BioMedica: Annual Revenues, 2014-2021 (GBP Million)
Figure 8.4 Sanofi: Annual Revenues, 2014-Q1 2022 (EUR Billion)
Figure 8.5 WuXi AppTec: Annual Revenues, 2017-Q1 2022 (RMB Million)
Figure 9.1 Company Competitiveness Analysis: Distribution of Players based in North America (In-House Manufacturers)
Figure 9.2 Company Competitiveness Analysis: Distribution of Players based in Europe (In-House Manufacturers)
Figure 9.3 Company Competitiveness Analysis: Distribution of Players based in North America (Contract Manufacturing Organizations)
Figure 9.4 Company Competitiveness Analysis: Distribution of Players based in Europe (Contract Manufacturing Organizations)
Figure 9.5 Company Competitiveness Analysis: Distribution of Players based in Asia-Pacific and Rest of the World (Contract Manufacturing Organizations)
Figure 9.6 Company Competitiveness Analysis: Distribution of Players based in North America (In-House and Contract Manufacturing Organizations)
Figure 9.7 Company Competitiveness Analysis: Distribution of Players based in Europe (In-House and Contract Manufacturing Organizations)
Figure 9.8 Company Competitiveness Analysis: Distribution of Players based in Asia-Pacific and Rest of the World (In-House and Contract Manufacturing Organizations)
Figure 10.1 Technology Competitiveness Analysis: Distribution of Players based in North America
Figure 10.2 Technology Competitiveness Analysis: Distribution of Players based in Europe and Asia-Pacific
Figure 11.1 Clinical Trial Analysis: Scope and Methodology
Figure 11.2 Clinical Trial Analysis: Cumulative Distribution by Trial Registration Year, Pre-2010-2022
Figure 11.3 Clinical Trial Analysis: Distribution by Trial Registration Year and Enrolled Patient Population, 2010-2022
Figure 11.4 Clinical Trial Analysis: Distribution by Trial Phase and Enrolled Patient Population
Figure 11.5 Clinical Trial Analysis: Distribution by Trial Status
Figure 11.6 Clinical Trial Analysis: Cumulative Year-wise Trend by Trial Status
Figure 11.7 Clinical Trial Analysis: Distribution by Study Design
Figure 11.8 Clinical Trial Analysis: Distribution by Therapeutic Area
Figure 11.9 Clinical Trial Analysis: Distribution by Target Disease Indication
Figure 11.10 Clinical Trial Analysis: Distribution by Type of Sponsor / Collaborator
Figure 11.11 Leading Industry Players: Distribution by Number of Registered Trials
Figure 11.12 Geographical Analysis: Distribution by Number of Registered Trials
Figure 11.13 Geographical Analysis: Distribution by Enrolled Patient Population
Figure 12.1 Adeno-Associated Viral Vector based Therapies: Cumulative Year-wise Trend, 2017-2022
Figure 12.2 Adeno-Associated Viral Vector based Therapies: Distribution by Type of Partnership, 2017-2022
Figure 12.3 Adeno-Associated Viral Vector based Therapies: Distribution by Year of Partnership and Type of Partner, 2017-2022
Figure 12.4 Adeno-Associated Viral Vector based Therapies: Distribution by Type of Partnership and Type of Partner, 2017-2022
Figure 12.5 Adeno-Associated Viral Vector based Therapies: Distribution by Therapeutic Area, 2017-2022
Figure 12.6 Most Active Players: Distribution by Number of Partnerships
Figure 12.7 Adeno-Associated Viral Vector based Therapies: Regional Distribution
Figure 12.8 Adeno-Associated Viral Vector based Therapies: Intercontinental and Intracontinental Agreements
Figure 12.9 Adeno-Associated Viral Vector Manufacturers: Cumulative Year-wise Trend, 2017-2022
Figure 12.10 Adeno-Associated Viral Vector Manufacturers: Distribution by Type of Partnership, 2017-2022
Figure 12.11 Adeno-Associated Viral Vector Manufacturers: Distribution by Year of Partnership and Type of Partner, 2017-2022
Figure 12.12 Adeno-Associated Viral Vector Manufacturers: Distribution by Type of Partnership and Type of Partner, 2017-2022
Figure 12.13 Adeno-Associated Viral Vector Manufacturers: Distribution by Therapeutic Area, 2017-2022
Figure 12.14 Most Active Players: Distribution by Number of Partnerships
Figure 12.15 Adeno-Associated Viral Vector Manufacturers: Regional Distribution
Figure 12.16 Adeno-Associated Viral Vector Manufacturers: Intercontinental and Intracontinental Agreements
Figure 14.1 Patent Analysis: Distribution by Type of Patent
Figure 14.2 Patent Analysis: Cumulative Distribution by Publication Year, 2017-2022
Figure 14.3 Patent Analysis: Cumulative Year-wise Distribution of Patent Applications, 2017-2022
Figure 14.4 Patent Analysis: Cumulative Distribution by Granted Patents, 2017-2022
Figure 14.5 Patent Analysis: Distribution by Geography
Figure 14.6 Patent Analysis: Distribution by CPC Symbols
Figure 14.7 Patent Analysis: Cumulative Year-wise Distribution by Type of Applicant
Figure 14.8 Leading Industry Players: Distribution by Number of Patents
Figure 14.9 Leading Individual Assignees: Distribution by Number of Patents
Figure 14.10 Patent Benchmarking: Distribution by Patent Characteristics (CPC Symbols)
Figure 14.11 Patent Valuation: Distribution of Patent by Age (2002-2022)
Figure 14.12 Patent Valuation: Distribution by Number of Citations
Figure 15.1 Start-up Health Indexing: Distribution by Location of Headquarters
Figure 15.2 Start-up Health Indexing: Distribution by Pipeline Strength
Figure 15.3 Start-up Health Indexing: Distribution by Pipeline Maturity
Figure 15.4 Start-up Health Indexing: Distribution by Indication Diversity
Figure 15.5 Start-up Health Indexing: Distribution by Number of Patents
Figure 15.6 Start-up Health Indexing: Distribution by Partnership Activity
Figure 15.7 Start-up Health Indexing
Figure 15.8 Leading Start-ups: Spider Web Analysis
Figure 16.1 Outsourcing: Go / No Go Framework
Figure 16.2 Outsourcing: Go / No Go Framework Methodology
Figure 16.3 Outsourcing: Go / No Go Framework for Adeno-Associated Viral Vector Developers
Figure 17.1 Adeno-Associated Viral Vector based Therapeutics Market, 2022-2035 (USD Billion)
Figure 17.2 Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Therapeutic Area, 2028 and 2035 (USD Billion)
Figure 17.3 Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Type of Therapy, 2028 and 2035 (USD Billion)
Figure 17.4 Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Type of Gene Delivery Method Used, 2028 and 2035 (USD Billion)
Figure 17.5 Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Route of Administration, 2028 and 2035 (USD Billion)
Figure 17.6 Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Geography, 2028 and 2035 (USD Billion)
Figure 17.7 Luxturna: Sales Forecast till 2035 (USD Million)
Figure 17.8 Zolgensma: Sales Forecast till 2035 (USD Million)
Figure 17.9 AGTC: Sales Forecast till 2035 (USD Million)
Figure 17.10 AMT-061: Sales Forecast till 2035 (USD Million)
Figure 17.11 BMN 270: Sales Forecast till 2035 (USD Million)
Figure 17.12 Lumevoq: Sales Forecast till 2035 (USD Million)
Figure 17.13 LYS-SAF302: Sales Forecast till 2035 (USD Million)
Figure 17.14 NFS-01: Sales Forecast till 2035 (USD Million)
Figure 17.15 OAV-101: Sales Forecast till 2035 (USD Million)
Figure 17.16 PF-06838435: Sales Forecast till 2035 (USD Million)
Figure 17.17 PF-06939926: Sales Forecast till 2035 (USD Million)
Figure 17.18 PF-07055480: Sales Forecast till 2035 (USD Million)
Figure 17.19 PTC-AADC: Sales Forecast till 2035 (USD Million)
Figure 17.20 RGX-314: Sales Forecast till 2035 (USD Million)
Figure 17.21 SPK-8011: Sales Forecast till 2035 (USD Million)
Figure 17.22 SPR-9001: Sales Forecast till 2035 (USD Million)
Figure 17.23 Adeno-Associated Viral Vector Manufacturers Market, 2022-2035 (USD Million)
Figure 17.24 Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Phase of Development (USD Million)
Figure 17.25 Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Therapeutic Area (USD Million)
Figure 17.26 Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Application Area (USD Million)
Figure 17.27 Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Key Geographical Regions (USD Million)
Figure 17.28 Adeno-Associated Viral Vector Manufacturing Market for Commercial Products, 2022-2035: Distribution by Therapeutic Area (USD Million)
Figure 17.29 Adeno-Associated Viral Vector Manufacturing Market for Commercial Products, 2022-2035: Distribution by Application Area (USD Million)
Figure 17.30 Adeno-Associated Viral Vector Manufacturing Market for Commercial Products, 2022-2035: Distribution by Key Geographical Regions (USD Million)
Figure 17.31 Adeno-Associated Viral Vector Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Phase of Development (USD Million)
Figure 17.32 Adeno-Associated Viral Vector Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Application Area (USD Million)
Figure 17.33 Adeno-Associated Viral Vector Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Key Geographical Regions (USD Million)
Figure 17.34 Adeno-Associated Viral Vector Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Type of Animal Model Used (USD Million)
Figure 17.35 Adeno-Associated Viral Vector Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Application Area (USD Million)
Figure 17.36 Adeno-Associated Viral Vector Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Key Geographical Regions (USD Million)
Figure 18.1 Concluding Remarks: Market Landscape of Adeno-Associated Viral Vector based Therapies
Figure 18.2 Concluding Remarks: Market Landscape of Adeno-Associated Viral Vector Manufacturers
Figure 18.3 Concluding Remarks: Market Landscape of Adeno-Associated Viral Vector Manufacturing Technologies
Figure 18.4 Concluding Remarks: Clinical Trial Analysis
Figure 18.5 Concluding Remarks: Partnerships and Collaborations
Figure 18.6 Concluding Remarks: Patent Analysis
Figure 18.7 Concluding Remarks: Market Sizing and Opportunity Analysis

List Of Tables
Table 4.1 Adeno-Associated Viral Vector based Therapy Market: Information on Developer, Phase of Development, Target Indication, Therapeutic Area(s), Type of Gene / Molecular Targeted, Vector Used, Gene Delivery Method Used, Route of Administration and Type of Therapy
Table 4.2 Heat Map Representation: Distribution by Type of Gene Delivery Method Used
Table 4.3 Adeno-Associated Viral Vector based Therapy Market: Information on Special Designation(s) Awarded
Table 4.4 List of Adeno-Associated Viral Vector based Therapy Developers
Table 5.1 Adeno-Associated Viral Vector Manufacturers: Information on Year of Establishment, Company Size, Headquarters and Type of Product(s) Manufactured
Table 5.2 Adeno-Associated Viral Vector Manufacturers: Information on Location of AAV Manufacturing Facilities, Scale of Operation and Type of Manufacturer
Table 5.3 Heat Map: Distribution by Location of Headquarters and Scale of Operation
Table 5.4 Adeno-Associated Viral Vector Manufacturers: Information on Application Area(s)
Table 6.1 Adeno-Associated Viral Vector Manufacturing Technologies: Information on Company, Type of Technology, Scale of Operation, Application Area(s), Therapeutic Area(s)
Table 6.2 List of Adeno-Associated Viral Vector Manufacturing Technology Developers
Table 7.1 Company Snapshot: Spark Therapeutics
Table 7.2 Luxturna: Status of Development
Table 7.3 Company Snapshot: Novartis
Table 7.4 Zolgensma: Status of Development
Table 7.5 AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.6 LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.7 NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.8 RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.9 AMT-061: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.10 OAV-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.11 PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.12 PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.13 PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.14 SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.15 SRP-9001: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.16 BMN 270: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.17 Lumevoq® (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 7.18 PTC-AADC: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
Table 8.1 Adeno-Associated Viral Vector Service Providers: List of Companies Profiled
Table 8.2 Abeona Therapeutics: Company Snapshot
Table 8.3 Abeona Therapeutics: Vector Manufacturing Related Capabilities
Table 8.4 Abeona Therapeutics: Recent Developments and Future Outlook
Table 8.5 Aldevron: Company Snapshot
Table 8.6 Aldevron: Vector Manufacturing Related Capabilities
Table 8.7 Aldevron: Recent Developments and Future Outlook
Table 8.8 Oxford BioMedica: Company Snapshot
Table 8.9 Oxford BioMedica: Vector Manufacturing Related Capabilities
Table 8.10 Oxford BioMedica: Recent Developments and Future Outlook
Table 8.11 Sanofi: Company Snapshot
Table 8.12 Sanofi: Vector Manufacturing Related Capabilities
Table 8.13 Sanofi: Recent Developments and Future Outlook
Table 8.14 WuXi AppTec: Company Snapshot
Table 8.15 WuXi AppTec: Vector Manufacturing Related Capabilities
Table 8.16 WuXi AppTec: Recent Developments and Future Outlook
Table 8.17 Yposkesi: Company Snapshot
Table 8.18 Yposkesi: Vector Manufacturing Related Capabilities
Table 8.19 Yposkesi: Recent Developments and Future Outlook
Table 12.1 Adeno-Associated Viral Vector based Therapies: List of Partnerships and Collaborations, 2017-2022
Table 12.2 Adeno-Associated Viral Vector Manufacturers: List of Partnerships and Collaborations, 2017-2022
Table 13.1 Adeno-Associated Viral Vector-based Therapy Manufacturers: Most Likely Partners
Table 13.2 Adeno-Associated Viral Vector-based Therapy Manufacturers: Likely Partners
Table 13.3 Adeno-Associated Viral Vector-based Therapy Manufacturers: Less Likely Partners
Table 13.4 Adeno-Associated Viral Vector-based Therapy Manufacturers: Unlikely Partners
Table 14.1 Patent Analysis: CPC Sections
Table 14.2 Patent Analysis: CPC Subclasses
Table 14.3 Patent Analysis: CPC Symbol Definitions
Table 14.4 Patent Analysis: Summary of Benchmarking Analysis
Table 14.5 Patent Analysis: Categorization based on Weighted Valuation Scores
Table 14.6 Patent Portfolio: List of Leading Patents (by Highest Relative Valuation)
Table 14.7 Patent Portfolio: List of Leading Patents (by Number of Citations)
Table 15.1 List of Start-ups Developing Adeno-Associated Viral Vectors
Table 17.1 Adeno-Associated Viral Vector Therapeutics Market: Expected Launch Year of Advanced Stage Drug Candidates
Table 17.2 Luxturna: Target Patient Population
Table 17.3 Luxturna: Net Present Value (USD Million)
Table 17.4 Luxturna: Value Creation Analysis (USD Million)
Table 17.5 Zolgensma: Target Patient Population
Table 17.6 Zolgensma: Net Present Value (USD Million)
Table 17.7 Zolgensma: Value Creation Analysis (USD Million)
Table 17.8 AGTC 501: Target Patient Population
Table 17.9 AGTC 501: Net Present Value (USD Million)
Table 17.10 AGTC 501: Value Creation Analysis (USD Million)
Table 17.11 AMT-061: Target Patient Population
Table 17.12 AMT-061: Net Present Value (USD Million)
Table 17.13 AMT-061: Value Creation Analysis (USD Million)
Table 17.14 BMN 270: Target Patient Population
Table 17.15 BMN 270: Net Present Value (USD Million)
Table 17.16 BMN 270: Value Creation Analysis (USD Million)
Table 17.17 Lumevoq: Target Patient Population
Table 17.18 Lumevoq: Net Present Value (USD Million)
Table 17.19 Lumevoq: Value Creation Analysis (USD Million)
Table 17.20 LYS-SAF302: Target Patient Population
Table 17.21 LYS-SAF302: Net Present Value (USD Million)
Table 17.22 LYS-SAF302: Value Creation Analysis (USD Million)
Table 17.23 NFS-01: Target Patient Population
Table 17.24 NFS-01: Net Present Value (USD Million)
Table 17.25 NFS-01: Value Creation Analysis (USD Million)
Table 17.26 OAV-101: Target Patient Population
Table 17.27 OAV-101: Net Present Value (USD Million)
Table 17.28 OAV-101: Value Creation Analysis (USD Million)
Table 17.29 PF-06838435: Target Patient Population
Table 17.30 PF-06838435: Net Present Value (USD Million)
Table 17.31 PF-06838435: Value Creation Analysis (USD Million)
Table 17.32 PF-06939926: Target Patient Population
Table 17.33 PF-06939926: Net Present Value (USD Million)
Table 17.34 PF-06939926: Value Creation Analysis (USD Million)
Table 17.35 PF-07055480: Target Patient Population
Table 17.36 PF-07055480: Net Present Value (USD Million)
Table 17.37 PF-07055480: Value Creation Analysis (USD Million)
Table 17.38 PTC-AADC: Target Patient Population
Table 17.39 PTC-AADC: Net Present Value (USD Million)
Table 17.40 PTC-AADC: Value Creation Analysis (USD Million)
Table 17.41 RGX-314: Target Patient Population
Table 17.42 RGX-314: Net Present Value (USD Million)
Table 17.43 RGX-314: Value Creation Analysis (USD Million)
Table 17.44 SPK-8011: Target Patient Population
Table 17.45 SPK-8011: Net Present Value (USD Million)
Table 17.46 SPK-8011: Value Creation Analysis (USD Million)
Table 17.47 SRP-9001: Target Patient Population
Table 17.48 SRP-9001: Net Present Value (USD Million)
Table 17.49 SRP-9001: Value Creation Analysis (USD Million)
Table 19.1 Boston College: Organization Snapshot
Table 19.2 University of Florida: Organization Snapshot
Table 20.1 Adeno-Associated Viral Vector Based Therapies: Distribution by Phase of Development
Table 20.2 Adeno-Associated Viral Vector Based Therapies: Distribution by Therapeutic Area(s)
Table 20.3 Adeno-Associated Viral Vector Based Therapies: Distribution by Phase of Development and Therapeutic Area
Table 20.4 Adeno-Associated Viral Vector Based Therapies: Distribution by Type of Gene / Molecule Targeted
Table 20.5 Adeno-Associated Viral Vector Based Therapies: Distribution by Type of Therapy
Table 20.6 Adeno-Associated Viral Vector Based Therapies: Distribution by Type of Gene Delivery Method Used
Table 20.7 Adeno-Associated Viral Vector Based Therapies: Distribution by Route of Administration
Table 20.8 Adeno-Associated Viral Vector Based Therapies: Distribution by Special Designation(s) Awarded
Table 20.9 Adeno-Associated Viral Vector Based Therapy Developers: Distribution by Year of Establishment
Table 20.10 Adeno-Associated Viral Vector Based Therapy Developers: Distribution by Company Size
Table 20.11 Adeno-Associated Viral Vector Based Therapy Developers: Distribution by Location of Headquarters (Region-wise)
Table 20.12 Leading Developers: Distribution by Number of Therapies in Pipeline
Table 20.13 Adeno-Associated Viral Vector Manufacturers: Distribution by Year of Establishment
Table 20.14 Adeno-Associated Viral Vector Manufacturers: Distribution by Company Size
Table 20.15 Adeno-Associated Viral Vector Manufacturers: Distribution by Location of Headquarters (Region-wise)
Table 20.16 Adeno-Associated Viral Vector Manufacturers: Distribution by Location of Headquarters (Country-wise)
Table 20.17 Adeno-Associated Viral Vector Manufacturers: Distribution by Type of Product(s) Manufactured
Table 20.18 Adeno-Associated Viral Vector Manufacturers: Distribution by Location of Vector Manufacturing Facilities (Region-wise)
Table 20.19 Adeno-Associated Viral Vector Manufacturers: Distribution by Location of Vector Manufacturing Facilities (Country-wise)
Table 20.20 Adeno-Associated Viral Vector Manufacturers: Distribution by Type of Manufacturer and Company Size
Table 20.21 Adeno-Associated Viral Vector Manufacturers: Distribution by Scale of Operation
Table 20.22 Adeno-Associated Viral Vector Manufacturers: Distribution by Application Area(s)
Table 20.23 Adeno-Associated Viral Vector Manufacturing Technologies: Distribution by Type of Technology
Table 20.24 Adeno-Associated Viral Vector Manufacturing Technologies: Distribution by Scale of Operation
Table 20.25 Adeno-Associated Viral Vector Manufacturing Technologies: Distribution by Application Area(s)
Table 20.26 Adeno-Associated Viral Vector Manufacturing Technologies: Distribution by Therapeutic Area(s)
Table 20.27 Adeno-Associated Viral Vector Technology Developers: Distribution by Year of Establishment
Table 20.28 Adeno-Associated Viral Vector Technology Developers: Distribution by Company Size
Table 20.29 Adeno-Associated Viral Vector Technology Developers: Distribution by Location of Headquarters (Region-wise)
Table 20.30 Adeno-Associated Viral Vector Technology: Distribution by Location of Headquarters (Country-wise)
Table 20.31 Adeno-Associated Viral Vector Technology Developers: Distribution by Company Size and Location of Headquarters
Table 20.32 Leading Developers: Distribution by Number of Technologies
Table 20.33 Abeona Therapeutics:Annual Revenues, 2016-Q1 2022 (USD Million)
Table 20.34 Danaher: Annual Revenues, 2014-Q1 2022 (USD Billion)
Table 20.35 Oxford BioMedica: Annual Revenues, 2014-2021 (GBP Million)
Table 20.36 Sanofi: Annual Revenues, 2014-Q1 2022 (EUR Billion)
Table 20.37 WuXi AppTec: Annual Revenues, 2017-Q1 2022 (RMB Million)
Table 20.38 Clinical Trial Analysis: Cumulative Distribution by Trial Registration Year, Pre-2010-2022
Table 20.39 Clinical Trial Analysis: Distribution by Trial Registration Year and Enrolled Patient Population, 2010-2022
Table 20.40 Clinical Trial Analysis: Distribution by Trial Phase and Enrolled Patient Population
Table 20.41 Clinical Trial Analysis: Distribution by Trial Status
Table 20.42 Clinical Trial Analysis: Cumulative Year-wise Trend by Trial Status
Table 20.43 Clinical Trial Analysis: Distribution by Study Design
Table 20.44 Clinical Trial Analysis: Distribution by Therapeutic Area
Table 20.45 Clinical Trial Analysis: Distribution by Target Disease Indication
Table 20.46 Clinical Trial Analysis: Distribution by Type of Sponsor / Collaborator
Table 20.47 Leading Industry Players: Distribution by Number of Registered Trials
Table 20.48 Geographical Analysis: Distribution by Number of Registered Trials
Table 20.49 Geographical Analysis: Distribution by Enrolled Patient Population
Table 20.50 Adeno-Associated Viral Vector based Therapies: Cumulative Year-wise Trend, 2017-2022
Table 20.51 Adeno-Associated Viral Vector based Therapies: Distribution by Type of Partnership, 2017-2022
Table 20.52 Adeno-Associated Viral Vector based Therapies: Distribution by Year of Partnership and Type of Partner, 2017-2022
Table 20.53 Adeno-Associated Viral Vector based Therapies: Distribution by Type of Partnership and Type of Partner, 2017-2022
Table 20.54 Adeno-Associated Viral Vector based Therapies: Distribution by Therapeutic Area, 2017-2022
Table 20.55 Most Active Players: Distribution by Number of Partnerships
Table 20.56 Adeno-Associated Viral Vector based Therapies: Regional Distribution
Table 20.57 Adeno-Associated Viral Vector based Therapies: Intercontinental and Intracontinental Agreements
Table 20.58 Adeno-Associated Viral Vector Manufacturers: Cumulative Year-wise Trend, 2017-2022
Table 20.59 Adeno-Associated Viral Vector Manufacturers: Distribution by Type of Partnership, 2017-2022
Table 20.60 Adeno-Associated Viral Vector Manufacturers: Distribution by Year of Partnership and Type of Partner, 2017-2022
Table 20.61 Adeno-Associated Viral Vector Manufacturers: Distribution by Type of Partnership and Type of Partner, 2017-2022
Table 20.62 Adeno-Associated Viral Vector Manufacturers: Distribution by Therapeutic Area, 2017-2022
Table 20.63 Most Active Players: Distribution by Number of Partnerships
Table 20.64 Adeno-Associated Viral Vector Manufacturers: Regional Distribution
Table 20.65 Adeno-Associated Viral Vector Manufacturers: Intercontinental and Intracontinental Agreements
Table 20.66 Patent Analysis: Distribution by Type of Patent
Table 20.67 Patent Analysis: Cumulative Distribution by Publication Year, 2017-2022
Table 20.68 Patent Analysis: Cumulative Year-wise Distribution of Patent Application(s), 2017-2022
Table 20.69 Patent Analysis: Cumulative Distribution by Granted Patents, 2017-2022
Table 20.70 Patent Analysis: Distribution by Geography
Table 20.71 Patent Analysis: Cumulative Year-wise Distribution by Type of Applicant
Table 20.72 Leading Industry Players: Distribution by Number of Patents
Table 20.73 Leading Individual Assignees: Distribution by Number of Patents
Table 20.74 Leading Industry Players: Benchmarking by Patent Characteristics (CPC Symbols)
Table 20.75 Patent Analysis: Distribution of Patent by Age (2002-2022)
Table 20.76 Start-up Health Indexing: Distribution by Location of Headquarters
Table 20.77 Start-up Health Indexing: Distribution by Pipeline Strength
Table 20.78 Start-up Health Indexing: Distribution by Pipeline Maturity
Table 20.79 Start-up Health Indexing: Distribution by Indication Diversity
Table 20.80 Start-up Health Indexing: Distribution by Number of Patents
Table 20.81 Start-up Health Indexing: Distribution by Partnership Activity
Table 20.82 Adeno-Associated Viral Vector based Therapeutics Market, 2022-2035 (USD Billion)
Table 20.83 Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Therapeutic Area, 2028 and 2035 (USD Billion)
Table 20.84 Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Type of Therapy, 2028 and 2035 (USD Billion)
Table 20.85 Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Type of Gene Delivery Method Used, 2028 and 2035 (USD Billion)
Table 20.86 Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Route of Administration, 2028 and 2035 (USD Billion)
Table 20.87 Adeno-Associated Viral Vector based Therapeutics Market: Distribution by Geography, 2028 and 2035 (USD Billion)
Table 20.88 Luxturna: Sales Forecast till 2035 (USD Million)
Table 20.89 Zolgensma: Sales Forecast till 2035 (USD Million)
Table 20.90 AGTC: Sales Forecast till 2035 (USD Million)
Table 20.91 AMT-061: Sales Forecast till 2035 (USD Million)
Table 20.92 BMN 270: Sales Forecast till 2035 (USD Million)
Table 20.93 Lumevoq: Sales Forecast till 2035 (USD Million)
Table 20.94 LYS-SAF302: Sales Forecast till 2035 (USD Million)
Table 20.95 NFS-01: Sales Forecast till 2035 (USD Million)
Table 20.96 OAV-101: Sales Forecast till 2035 (USD Million)
Table 20.97 PF-06838435: Sales Forecast till 2035 (USD Million)
Table 20.98 PF-06939926: Sales Forecast till 2035 (USD Million)
Table 20.99 PF-07055480: Sales Forecast till 2035 (USD Million)
Table 20.100 PTC-AADC: Sales Forecast till 2035 (USD Million)
Table 20.101 RGX-314: Sales Forecast till 2035 (USD Million)
Table 20.102 SPK-8011: Sales Forecast till 2035 (USD Million)
Table 20.103 SPR-9001: Sales Forecast till 2035 (USD Million)
Table 20.104 Adeno-Associated Viral Vector Manufacturers Market, 2022-2035 (USD Million)
Table 20.105 Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Phase of Development (USD Million)
Table 20.106 Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Therapeutic Area (USD Million)
Table 20.107 Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Application Area (USD Million)
Table 20.108 Adeno-Associated Viral Vector Manufacturing Market, 2022-2035: Distribution by Key Geographical Regions (USD Million)
Table 20.109 Adeno-Associated Viral Vector Manufacturing Market for Commercial Products, 2022-2035: Distribution by Therapeutic Area (USD Million)
Table 20.110 Adeno-Associated Viral Vector Manufacturing Market for Commercial Products, 2022-2035: Distribution by Application Area (USD Million)
Table 20.111 Adeno-Associated Viral Vector Manufacturing Market for Commercial Products, 2022-2035: Distribution by Key Geographical Regions (USD Million)
Table 20.112 Adeno-Associated Viral Vector Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Phase of Development (USD Million)
Table 20.113 Adeno-Associated Viral Vector Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Application Area (USD Million)
Table 20.114 Adeno-Associated Viral Vector Manufacturing Market for Clinical Candidates, 2022-2035: Distribution by Key Geographical Regions (USD Million)
Table 20.115 Adeno-Associated Viral Vector Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Type of Animal Model Used (USD Million)
Table 20.116 Adeno-Associated Viral Vector Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Application Area (USD Million)
Table 20.117 Adeno-Associated Viral Vector Manufacturing Market for Preclinical Candidates, 2022-2035: Distribution by Key Geographical Regions (USD Million)

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • 4D Molecular Therapeutics
  • AAVantgarde Bio
  • AavantiBio
  • aaVective
  • AaviGen
  • AAVogen
  • AbbVie
  • Abeona Therapeutics
  • Addgene
  • Adrenas Therapeutics
  • Advanced BioScience Laboratories
  • Adverum Biotechnologies
  • Aevitas Therapeutics
  • Affinia Therapeutics
  • Agathos Biologics
  • Applied Genetics Technologies Corporation
  • Akouos
  • Aldevron
  • Allergan
  • AllStripes
  • Alzheimer’s Drug Discovery Foundation
  • Ambulero
  • Amicus Therapeutics
  • Amphastar Pharmaceuticals
  • AMSBIO
  • Andelyn Biosciences
  • Anlong Biopharmaceutical
  • Applied Biological Materials
  • Applied Viromics
  • Arbor Biotechnologies
  • Aruvant Sciences
  • ASC Therapeutics
  • Asklepios BioPharmaceutical
  • Aspa Therapeutics
  • Astellas Gene Therapies
  • Astellas Pharma
  • Atamyo Therapeutics
  • Atsena Therapeutics
  • ATVIO Biotechnology
  • Auburn University
  • Audentes Therapeutics
  • Audition Therapeutics
  • AveXis (acquired by Novartis)
  • AviadoBio
  • Avid Bioservices
  • Axovia Therapeutics
  • Batavia Biosciences
  • Bayer
  • Beam Therapeutics
  • Belief Biomed
  • Biogen
  • Biointaxis
  • BioMarin Pharmaceutical
  • Biomay
  • Bionic Sight
  • Biovian
  • Blue Sky BioServices
  • Borea Therapeutics
  • Brain Neurotherapy Bio (an integrated unit of AskBio)
  • BridgeBio
  • Bristol Myers Squibb
  • California Institute for Regenerative Medicine
  • Canadian Institutes of Health Research
  • Canadian Respiratory Research Network (CRRN)
  • CANbridge Pharmaceuticals
  • CanSino Biologics
  • Capsida Biotherapeutics
  • Case Western Reserve University
  • Catalent Biologics
  • Catalyst Biosciences
  • CavoGene LifeSciences
  • Cellular Biomedicine Group
  • Celonic
  • The Center for Breakthrough Medicines (CBM) 
  • Center for Process Innovation (CPI)
  • CEVEC Pharmaceuticals
  • Charcot-Marie-Tooth Association (CMTA)
  • University of Navarra
  • CLINO
  • Coave Therapeutics
  • Cobra Biologics
  • CODA Biotherapeutics
  • Columbia University
  • CombiGene
  • Creative Biogene
  • Creative Biolabs
  • CRISPR Therapeutics
  • CSL Behring
  • Cyprium Therapeutics
  • Cystic Fibrosis Foundation
  • Cytiva
  • Daiichi Sankyo
  • Decibel Therapeutics
  • DEKK-TEC
  • DiNAQOR
  • Duke University
  • Dyno Therapeutics
  • Editas Medicine
  • ElevateBio
  • Eli Lilly
  • Emas Pharma
  • Emory University
  • Encoded Therapeutics
  • Endsulin
  • Esteve
  • ExcellGene
  • Excision BioTherapeutics
  • Exegenesis Bio
  • Exothera
  • Expression Therapeutics
  • FinVector
  • Forge Biologics
  • Freeline Therapeutics
  • FUJIFILM Diosynth Biotechnologies
  • Gene Therapy Research Institution
  • GeneCopoeia
  • GeneDetect
  • Genethon
  • Genezen
  • Genflow Biosciences
  • GenIbet Biopharmaceuticals
  • Genprex
  • GenSight Biologics
  • Genzyme
  • Gyroscope Therapeutics
  • Hamilton Health Sciences
  • Harvard University
  • Helixmith
  • Roche
  • Homology Medicines
  • Huida (Shanghai) Biotechnology
  • iECURE
  • Innovavector
  • Inspirna (formetly known as Rgenix)
  • Institut de Myologie
  • Intas Pharmaceuticals
  • Intellia Therapeutics
  • IVERIC bio
  • Jaguar Gene Therapy
  • Janssen Pharmaceuticals
  • Kate Therapeutics
  • Kite Pharma
  • Kriya Therapeutics
  • Krystal Biotech
  • Kubota Vision
  • Lacerta Therapeutics
  • Leiden University Medical Center
  • Lentigen Technology (a subsidiary of Miltenyi Biotec)
  • LEXEO Therapeutics
  • Lipigon Pharmaceuticals
  • LogicBio Therapeutics
  • Lonza
  • Lysogene
  • Massachusetts General Hospital
  • MaxCyte
  • McMaster University
  • MeiraGTx
  • Milo Biotechnology
  • Mirum Pharmaceuticals
  • MolMed
  • Myrtelle
  • NanoCor Therapeutics
  • Nanoscope Therapeutics
  • National Institute of Child Health and Human Development
  • National Institute of Health
  • Nationwide Children’s Hospital
  • Nature Technology
  • NeuExcell Therapeutics
  • NeuGATE Theranostics
  • Neuracel Genetics
  • Neurocrine Biosciences
  • Neurogastrx
  • Neurogene
  • NeuroLogix
  • Neurophth Therapeutics
  • Nuevocor
  • Nikon CeLL innovation
  • Ninevah Therapeutics
  • North Shore University Hospital
  • Novartis
  • Novartis Gene Therapies (formerly known as AveXis)
  • Novasep
  • Ocugen
  • Odylia Therapeutics
  • Lung Health Foundation
  • Opus Genetics
  • Orchard Therapeutics
  • Oregon Health and Science University
  • Otonomy
  • Oxford BioMedica
  • OXGENE
  • Pall Biotech
  • Paragon Bioservices (acquired by Catalent Biologics)
  • Passage Bio
  • PeriphaGen
  • Pfizer
  • Phoenix Nest
  • PlasmidFactory
  • Poseida Therapeutics
  • PRA Health Sciences
  • Precigen
  • Precision BioSciences
  • Prevail Therapeutics
  • ProBioGen
  • ProMab Biotechnologies
  • PTC Therapeutics
  • Purespring Therapeutics
  • Redpin Therapeutics
  • ReflectionBio
  • Regeneron Pharmaceuticals
  • REGENXBIO
  • ReiThera
  • Renova Therapeutics
  • Reyon Pharmaceutical
  • Roche
  • Rocket Pharmaceuticals
  • Rubius Therapeutics
  • SAB Technology
  • BioReliance
  • Sangamo Therapeutics
  • Sanofi
  • Sarepta Therapeutics
  • Selecta Biosciences
  • Sensorion
  • Shape Therapeutics
  • Sibiono GeneTech
  • SignaGen Laboratories
  • Sio Gene Therapies (formerly known as Axovant Gene Therapies)
  • SIRION Biotech
  • Skyline Therapeutics
  • Solid Biosciences
  • SparingVision
  • Spark Therapeutics
  • Spirovant Sciences
  • SpliceBio
  • SQZ Biotechnologies
  • St. Joseph’s Healthcare Hamilton
  • Stockholm University
  • StrideBio
  • SwanBio Therapeutics
  • Syne qua non
  • System Biosciences
  • Takeda Pharmaceutical
  • Tamid Bio
  • Taysha Gene Therapies
  • TeamOn
  • Canadian Frailty Network (CFN)
  • Temple University
  • Tenaya Therapeutics
  • Tetraneuron
  • Nationwide Children's Hospital
  • Théa Open Innovation
  • Theravectys
  • Thermo Fisher Scientific
  • Tigris Pharmaceuticals
  • ToolGen
  • Universitat Autònoma de Barcelona  
  • UCB Biopharma
  • Ultragenyx
  • UMass Chan Medical School
  • The University of North Carolina
  • uniQure
  • University College London
  • University Hospitals Cleveland Medical Center
  • The University of Alabama at Birmingham
  • University of California
  • University of Florida
  • University of Massachusetts
  • University of North Carolina
  • University of Pennsylvania
  • University of Pittsburgh
  • University of Washington School of Medicine
  • Usher 2020 Foundation
  • VBL Therapeutics
  • Vector Biolabs
  • VectorY
  • Vertex Pharmaceuticals
  • Vibalogics
  • Vigene Biosciences (acquired by Charles River Laboratories)
  • ViGeneron
  • ViralGEN
  • ViraQuest
  • Virovek
  • VIVEbiotech
  • Vivet Therapeutics
  • Voyager Therapeutics
  • Weill Cornell Medical College
  • Weizmann Institute of Science
  • WuXi Advanced Therapies
  • Yposkesi

Methodology

 

 

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