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, Hematological 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

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Overview

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?

Contents

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: Roots Analysis Perspective

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 DATA

21. 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: Roots Analysis Perspective
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)

List Of Companies

  1. 4D Molecular Therapeutics
  2. AAVantgarde Bio
  3. AavantiBio
  4. aaVective
  5. AaviGen
  6. AAVogen
  7. AbbVie
  8. Abeona Therapeutics
  9. Addgene
  10. Adrenas Therapeutics
  11. Advanced BioScience Laboratories
  12. Adverum Biotechnologies
  13. Aevitas Therapeutics
  14. Affinia Therapeutics
  15. Agathos Biologics
  16. Applied Genetics Technologies Corporation
  17. Akouos
  18. Aldevron
  19. Allergan
  20. AllStripes
  21. Alzheimer’s Drug Discovery Foundation
  22. Ambulero
  23. Amicus Therapeutics
  24. Amphastar Pharmaceuticals
  25. AMSBIO
  26. Andelyn Biosciences
  27. Anlong Biopharmaceutical
  28. Applied Biological Materials
  29. Applied Viromics
  30. Arbor Biotechnologies
  31. Aruvant Sciences
  32. ASC Therapeutics
  33. Asklepios BioPharmaceutical
  34. Aspa Therapeutics
  35. Astellas Gene Therapies
  36. Astellas Pharma
  37. Atamyo Therapeutics
  38. Atsena Therapeutics
  39. ATVIO Biotechnology
  40. Auburn University
  41. Audentes Therapeutics
  42. Audition Therapeutics
  43. AveXis (acquired by Novartis)
  44. AviadoBio
  45. Avid Bioservices
  46. Axovia Therapeutics
  47. Batavia Biosciences
  48. Bayer
  49. Beam Therapeutics
  50. Belief Biomed
  51. Biogen
  52. Biointaxis
  53. BioMarin Pharmaceutical
  54. Biomay
  55. Bionic Sight
  56. Biovian
  57. Blue Sky BioServices
  58. Borea Therapeutics
  59. Brain Neurotherapy Bio (an integrated unit of AskBio)
  60. BridgeBio
  61. Bristol Myers Squibb
  62. California Institute for Regenerative Medicine
  63. Canadian Institutes of Health Research
  64. Canadian Respiratory Research Network (CRRN)
  65. CANbridge Pharmaceuticals
  66. CanSino Biologics
  67. Capsida Biotherapeutics
  68. Case Western Reserve University
  69. Catalent Biologics
  70. Catalyst Biosciences
  71. CavoGene LifeSciences
  72. Cellular Biomedicine Group
  73. Celonic
  74. The Center for Breakthrough Medicines (CBM) 
  75. Center for Process Innovation (CPI)
  76. CEVEC Pharmaceuticals
  77. Charcot-Marie-Tooth Association (CMTA)
  78. University of Navarra
  79. CLINO
  80. Coave Therapeutics
  81. Cobra Biologics
  82. CODA Biotherapeutics
  83. Columbia University
  84. CombiGene
  85. Creative Biogene
  86. Creative Biolabs
  87. CRISPR Therapeutics
  88. CSL Behring
  89. Cyprium Therapeutics
  90. Cystic Fibrosis Foundation
  91. Cytiva
  92. Daiichi Sankyo
  93. Decibel Therapeutics
  94. DEKK-TEC
  95. DiNAQOR
  96. Duke University
  97. Dyno Therapeutics
  98. Editas Medicine
  99. ElevateBio
  100. Eli Lilly
  101. Emas Pharma
  102. Emory University
  103. Encoded Therapeutics
  104. Endsulin
  105. Esteve
  106. ExcellGene
  107. Excision BioTherapeutics
  108. Exegenesis Bio
  109. Exothera
  110. Expression Therapeutics
  111. FinVector
  112. Forge Biologics
  113. Freeline Therapeutics
  114. FUJIFILM Diosynth Biotechnologies
  115. Gene Therapy Research Institution
  116. GeneCopoeia
  117. GeneDetect
  118. Genethon
  119. Genezen
  120. Genflow Biosciences
  121. GenIbet Biopharmaceuticals
  122. Genprex
  123. GenSight Biologics
  124. Genzyme
  125. Gyroscope Therapeutics
  126. Hamilton Health Sciences
  127. Harvard University
  128. Helixmith
  129. Roche
  130. Homology Medicines
  131. Huida (Shanghai) Biotechnology
  132. iECURE
  133. Innovavector
  134. Inspirna (formetly known as Rgenix)
  135. Institut de Myologie
  136. Intas Pharmaceuticals
  137. Intellia Therapeutics
  138. IVERIC bio
  139. Jaguar Gene Therapy
  140. Janssen Pharmaceuticals
  141. Kate Therapeutics
  142. Kite Pharma
  143. Kriya Therapeutics
  144. Krystal Biotech
  145. Kubota Vision
  146. Lacerta Therapeutics
  147. Leiden University Medical Center
  148. Lentigen Technology (a subsidiary of Miltenyi Biotec)
  149. LEXEO Therapeutics
  150. Lipigon Pharmaceuticals
  151. LogicBio Therapeutics
  152. Lonza
  153. Lysogene
  154. Massachusetts General Hospital
  155. MaxCyte
  156. McMaster University
  157. MeiraGTx
  158. Milo Biotechnology
  159. Mirum Pharmaceuticals
  160. MolMed
  161. Myrtelle
  162. NanoCor Therapeutics
  163. Nanoscope Therapeutics
  164. National Institute of Child Health and Human Development
  165. National Institute of Health
  166. Nationwide Children’s Hospital
  167. Nature Technology
  168. NeuExcell Therapeutics
  169. NeuGATE Theranostics
  170. Neuracel Genetics
  171. Neurocrine Biosciences
  172. Neurogastrx
  173. Neurogene
  174. NeuroLogix
  175. Neurophth Therapeutics
  176. Nuevocor
  177. Nikon CeLL innovation
  178. Ninevah Therapeutics
  179. North Shore University Hospital
  180. Novartis
  181. Novartis Gene Therapies (formerly known as AveXis)
  182. Novasep
  183. Ocugen
  184. Odylia Therapeutics
  185. Lung Health Foundation
  186. Opus Genetics
  187. Orchard Therapeutics
  188. Oregon Health and Science University
  189. Otonomy
  190. Oxford BioMedica
  191. OXGENE
  192. Pall Biotech
  193. Paragon Bioservices (acquired by Catalent Biologics)
  194. Passage Bio
  195. PeriphaGen
  196. Pfizer
  197. Phoenix Nest
  198. PlasmidFactory
  199. Poseida Therapeutics
  200. PRA Health Sciences
  201. Precigen
  202. Precision BioSciences
  203. Prevail Therapeutics
  204. ProBioGen
  205. ProMab Biotechnologies
  206. PTC Therapeutics
  207. Purespring Therapeutics
  208. Redpin Therapeutics
  209. ReflectionBio
  210. Regeneron Pharmaceuticals
  211. REGENXBIO
  212. ReiThera
  213. Renova Therapeutics
  214. Reyon Pharmaceutical
  215. Roche
  216. Rocket Pharmaceuticals
  217. Rubius Therapeutics
  218. SAB Technology
  219. BioReliance
  220. Sangamo Therapeutics
  221. Sanofi
  222. Sarepta Therapeutics
  223. Selecta Biosciences
  224. Sensorion
  225. Shape Therapeutics
  226. Sibiono GeneTech
  227. SignaGen Laboratories
  228. Sio Gene Therapies (formerly known as Axovant Gene Therapies)
  229. SIRION Biotech
  230. Skyline Therapeutics
  231. Solid Biosciences
  232. SparingVision
  233. Spark Therapeutics
  234. Spirovant Sciences
  235. SpliceBio
  236. SQZ Biotechnologies
  237. St. Joseph’s Healthcare Hamilton
  238. Stockholm University
  239. StrideBio
  240. SwanBio Therapeutics
  241. Syne qua non
  242. System Biosciences
  243. Takeda Pharmaceutical
  244. Tamid Bio
  245. Taysha Gene Therapies
  246. TeamOn
  247. Canadian Frailty Network (CFN)
  248. Temple University
  249. Tenaya Therapeutics
  250. Tetraneuron
  251. Nationwide Children's Hospital
  252. Théa Open Innovation
  253. Theravectys
  254. Thermo Fisher Scientific
  255. Tigris Pharmaceuticals
  256. ToolGen
  257. Universitat Autònoma de Barcelona  
  258. UCB Biopharma
  259. Ultragenyx
  260. UMass Chan Medical School
  261. The University of North Carolina
  262. uniQure
  263. University College London
  264. University Hospitals Cleveland Medical Center
  265. The University of Alabama at Birmingham
  266. University of California
  267. University of Florida
  268. University of Massachusetts
  269. University of North Carolina
  270. University of Pennsylvania
  271. University of Pittsburgh
  272. University of Washington School of Medicine
  273. Usher 2020 Foundation
  274. VBL Therapeutics
  275. Vector Biolabs
  276. VectorY
  277. Vertex Pharmaceuticals
  278. Vibalogics
  279. Vigene Biosciences (acquired by Charles River Laboratories)
  280. ViGeneron
  281. ViralGEN
  282. ViraQuest
  283. Virovek
  284. VIVEbiotech
  285. Vivet Therapeutics
  286. Voyager Therapeutics
  287. Weill Cornell Medical College
  288. Weizmann Institute of Science
  289. WuXi Advanced Therapies
  290. Yposkesi

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