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Gene Editing beyond CRISPR Market: Focus on Zinc Finger Nucleases (ZFNs), Transcription Activator-Like Effector Nucleases (TALENs) and Meganucleases Edited Therapies: Distribution by Type of Payment (Upfront and Milestone Payment) and Distribution by Geography (North America, Europe, Asia-Pacific and Rest of the World): Industry Trends and Global Forecasts, 2022-2035

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  • Published
    January 2022

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    180

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    350

Example Insights

Gene-Editing-beyond-CRISPR-Market-Context Gene-Editing-beyond-CRISPR-Market-List-of-non-CRISPR Gene-Editing-beyond-CRISPR-Market-Year-of-Establishment Gene-Editing-beyond-CRISPR-Market-Publication-Year
Gene-Editing-beyond-CRISPR-Market-Patent-Analysis Gene-Editing-beyond-CRISPR-Market-Partnerships-and-Collaborations Gene-Editing-beyond-CRISPR-Market-Pipeline Gene-Editing-beyond-CRISPR-Market-Academic-Grant-Analysis
Gene-Editing-beyond-CRISPR-Market-Funding-and-Investments Gene-Editing-beyond-CRISPR-Market-Licensing-Deals    

 

Overview

Isolation of the first site-specific restriction enzyme, Hind II, in the 1970s, became one of the breakthrough advances in biotechnology. This led to the discovery of various methods for manipulating living creatures at the genomic level, thereby, opening up a slew of new possibilities in basic and applied life sciences domain. Soon after, in the 1980s, the US FDA authorized human insulin, marketed under the brand name HUMULIN®, as the world's first genetically modified medication. With the development of various DNA modulation technologies, such as zinc-finger nucleases (ZFNs), TAL effector nucleases (TALENs), engineered endonucleases / meganucleases (EMNs) and clustered regularly interspaced short palindromic repeats (CRISPR), genetic engineering and genome editing concepts have gained significant attention over the last two decades. In fact, there have been several advancements in the field of genome editing, which provide investigators the ability to introduce sequence-specific modifications into the genomes of a broad spectrum of cell types and organisms.

Several medical researchers and industry stakeholders are presently engaged in exploring the potential of different gene editing technologies for basic research and development of gene editing solutions. However, the therapeutic use of these versatile genetic manipulation tools is only being investigated by selective stakeholders in the pharmaceutical and biotechnology sector. This can be attributed to the implementation of surrogate licensing model, which has granted exclusive control of the associated intellectual property (IP) to the drug developers. Clinical trials of ZFNs, TALENs and meganuclease based therapeutics are primarily focused on infectious diseases and oncological disorders; however, several product candidates against certain hematological disorders, genetic disorders and neurological disorders are being evaluated in discovery and preclinical stages of development. Over time, a number of industry and non-industry players have also been validating the therapeutic applications of these technologies, which has, in turn, prompted the establishment of strategic partnerships. In fact, the growing popularity of such technologies has attracted an investment worth USD 2 billion into companies engaged in this field of research. Promising clinical results, and ongoing technical developments, coupled with the growing interest of biopharmaceutical developers, are anticipated to push the pipeline products to higher phases of development. We believe that the market is likely to evolve at a sustained pace over the next decade.

Scope of the Report

The “Gene Editing beyond CRISPR Market: Focus on Zinc Finger Nucleases (ZFNs), Transcription Activator-Like Effector Nucleases (TALENs) and Meganucleases Edited Therapies: Distribution by Type of Payment (Upfront and Milestone Payment) and Distribution by Geography (North America, Europe, Asia-Pacific 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 of the ZFNs, TALENs and meganucleases based therapies market, in the mid to long term. The study underlines an in-depth analysis, highlighting the diverse capabilities of stakeholders engaged in this domain. Amongst other elements, the report includes:

  • A detailed assessment of the current market landscape of ZFNs based therapies, based on several relevant parameters, such as phase of development (clinical and preclinical), biological target (BCL 11A, C9ORF72, CD 19, HLA-A2, HTT gene, MAPT, SNCA gene, VEGF-A, HPV16/E7 and HPV18/E7), delivery vehicle (non-viral and naked plasmid), target indication, therapeutic area (blood disorders, infectious diseases, neurological disorders, oncological disorders and others) and type of therapy (in-vivo and ex-vivo). In addition, it provides details on the companies engaged in the development of ZFNs based therapies, along with information on their year of establishment, location of headquarters and company size.
  • A detailed assessment of the current market landscape of TALENs based therapies, based on several relevant parameters, such as phase of development (clinical and preclinical), target indication, type of cells (T Cells, NK Cells, HPV positive and B Cells), biological target (iNK, CD19, BCMA, TCR-α/CD52, CD22, CD38, CS1, GPCR, HPV18/E7, HPV16/E6, HPV16/E7 and HPV18/E6), cell / gene therapy (NK-Cell Therapy, T-Cell Therapy and CAR-T), type of therapy (in-vivo and ex-vivo), gene editing approach (insertion and deletion) and route of administration (intravenous, topical and intravaginal). In addition, it provides details on the companies engaged in the development of TALEN based therapies, along with information on their year of establishment, company size and location of headquarters.
  • A detailed assessment of the current market landscape of Meganucleases based therapies, based on several relevant parameters, such as phase of development (clinical and preclinical), gene editing approach, delivery vehicle (AAV9 and lentiviral), therapeutic area(s) (genetic disorders, liver disorders, kidney disorders, rare disorders, ocular disorders and oncological disorders), route of administration, type of cells, type of therapy and biological target(s) (HA01, TTR, PCSK9 and ApoC3). In addition, it provides details on the companies engaged in the development of meganucleases based therapies, along with information on their year of establishment, company size and location of headquarters.
  • Detailed profiles of prominent players engaged in the development of ZFNs, TALENs and meganucleases based therapies. Each profile features a brief overview of the company, details on its drug candidates, its financial information (if available), recent developments and an informed future outlook.
  • A detailed analysis of completed and ongoing clinical trials of various ZFNs, TALENs and meganucleases based therapies, based on different parameters, such as trial status, trial registration year, target disease indication, trial phase, study design, number of patients enrolled and type of sponsor / collaborator. In addition, the chapter highlights the active industry players and location of the trials.
  • A detailed review of around 1,250 peer-reviewed, scientific articles related to research on ZFNs, TALENs and meganucleases based therapies on the basis of several parameters, such as year of publication, therapeutic area, emerging focus area. The chapter also highlights the top journals and top authors (in terms of number of articles published).
  • An in-depth analysis of academic grants that have been awarded to various research institutes for projects related to ZFNs, TALENs and meganucleases based therapies, during the period, 2017-2021, based on parameters, such as year of grant award, amount awarded, type of funding institute center, activity code, support period, prominent program officers, emerging focus areas and type of grant application. Further, the chapter also highlights the popular recipient organizations, (in terms of number of grants and amount awarded), study section, amount awarded and purpose of grant, and location of recipients.
  • An analysis of the partnerships that have been established in this domain till 2021, covering instances of R&D agreements, clinical trial agreements, mergers / acquisitions, product development and commercialization agreements, licensing agreements, asset acquisitions and product development and manufacturing agreements. 
  • A detailed analysis of the various investments made till 2021, including grants, seed financing, venture capital financing, IPOs, secondary offering, other equity, post IPO equity and equity crowdfunding in the companies focused on the development of therapies based on ZFNs, TALENs and meganucleases.
  • An insightful analysis of the patents filed / granted for ZFNs, TALENs and meganucleases till 2021, taking into consideration various parameters, such as type of patent, issuing authority / patent offices involved, Cooperative Patent Classification (CPC) symbols, type of company, leading industry and non-industry players (on the basis of number of patents) and individual patent assignees (in terms of size of intellectual property portfolio).
  • A list of key opinion leaders (KOLs) within this domain, and their assessment (based on the strength and activeness) represented in the form of 2×2 matrices. The chapter also includes an analysis evaluating the (relative) level of expertise of different KOLs, based on number of publications, number of citations, participation in clinical trials, number of affiliations and strength of professional network.
  • A case study on the CRISPR / Cas based therapies that are currently in different stages of development. The chapter features a detailed analysis of pipeline molecules, based on various parameters, such as target therapeutic area (autoimmune disorders, cardiovascular disorders, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscular diseases, neurological disorders, oncological disorders, ophthalmic diseases and others), phase of development (discovery, preclinical and clinical), approach of therapy (ex vivo and in vivo), cell source (autologous and allogeneic), type of therapy (CAR-T therapy, HSC therapy, T cell therapy, Phage therapy and others), and the type of technology used. 

One of the key objectives of the report was to estimate the existing market size and identify potential future growth opportunities of novel technologies for the development of ZFNs, TALENs and meganucleases based therapies in the mid to long term. Based on the likely licensing deal structures and agreements that are expected to be signed in the foreseen future, we have provided informed estimates on the evolution of the market over the period 2021-2035. The future opportunity within the gene editing market has been segmented across [A] type of payment (upfront payment and milestone payment) and [B] geography (North America, Europe, and Asia Pacific 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.

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

  • Who are the players engaged in the development of gene editing therapies beyond CRISPR?
  • Which are the key drugs being developed across early and late stages of development?
  • Which companies are actively involved in conducting clinical trials for ZFNs, TALENs and meganucleases based therapies?
  • What is the focus of various publications related to ZFNs, TALENs and meganucleases based therapies?
  • Which are the leading administering institute centers supporting the research related to gene editing market beyond CRISPR?
  • What kind of partnership models are commonly adopted by industry stakeholders?
  • What is the trend of capital investments in the gene editing beyond CRISPR market?
  • How has the intellectual property landscape in this market evolved over the years?
  • How is the current and future opportunity, related to ZFNs, TALENs and meganucleases based therapies, likely to be distributed across key market segments?

Contents

Chapter Outlines

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

Chapter 3 provides a general introduction to gene editing, evolution of genome editing tools and various types of genome editing tools. In addition, the chapter provides detailed information on the ZFNs, TALENs and meganucleases along with their structure and mechanism of action. Further, the chapter presents details on the various advantages and challenges associated with these gene editing tools and the future perspectives.

Chapter 4 provides an overview of the current market landscape of ZFNs, TALENs and meganucleases based therapies, including analyses based on several relevant parameters, such as phase of development, biological target, delivery vehicle, target indication, therapeutic area, type of therapy, cell / gene therapy, gene editing approach, delivery vehicle and route of administration. In addition, it provides details on the companies engaged in the development of ZFNs, TALENs and meganucleases based therapies, along with information on their year of establishment, company size and location of headquarters.

Chapter 5 includes detailed profiles of prominent players engaged in the development of ZFNs, TALENs and meganucleases based therapies. Each profile features a brief overview of the company, details on its drug candidates, its financial information (if available), recent developments and an informed future outlook.

Chapter 6 provides information on completed and ongoing clinical studies of ZFNs, TALENs and meganucleases based therapies, based on different parameters, such as trial status, trial registration year, target disease indication, trial phase, study design, number of patients enrolled and type of sponsor / collaborator. In addition, the chapter highlights the active industry players and location of the trials.

Chapter 7 features a detailed review of peer-reviewed, scientific articles related to research on ZFNs, TALENs and meganucleases based therapies, on the basis of several parameters, such as year of publication, therapeutic area, emerging focus area. The chapter also highlights the top journals and top authors (in terms of number of articles published).

Chapter 8 provides an in-depth analysis of academic grants that have been awarded to various research institutes for projects related to ZFNs, TALENs and meganucleases based therapies, during the period, 2017- 2021, based on parameters, such as year of grant award, amount awarded, type of funding institute center, activity code, support period, prominent program officers, emerging focus areas and type of grant application. Further, the chapter also highlights the popular recipient organizations, (in terms of number of grants and amount awarded), study section, amount awarded and purpose of grant, and location of recipients.

Chapter 9 features an analysis of the partnerships that have been established in this domain, till 2021, covering instances of R&D agreements, clinical trial agreements, mergers / acquisitions, product development and commercialization agreements, licensing agreements, asset acquisitions and product development and manufacturing agreements. 

Chapter 10 presents a review of the funding and investments in this domain till 2021, including grants, seed financing, venture capital financing, IPOs, secondary offering, other equity, post IPO equity and equity crowdfunding. The chapter also includes a detailed analysis of funding and investment, based on several relevant parameters. 

Chapter 11 provides an insightful analysis of the patents filed / granted for ZFNs, TALENs and meganucleases till 2021, taking into consideration various parameters, such as type of patent, issuing authority / patent offices involved, Cooperative Patent Classification (CPC) symbols, emerging areas (in terms of number of patents filed / granted), type of company, leading industry and non-industry players (on the basis of number of patents) and individual patent assignees (in terms of size of intellectual property portfolio).

Chapter 12 presents a list of key opinion leaders (KOLs) within this domain, and their assessment (based on the strength and activeness) represented in the form of 2×2 matrices. The chapter also includes an analysis evaluating the (relative) level of expertise of different KOLs, based on number of publications, number of citations, participation in clinical trials, number of affiliations and strength of professional network.

Chapter 13 presents a case study on the CRISPR / Cas based therapeutics that are currently in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as target therapeutic area (autoimmune disorders, cardiovascular disorders, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscular diseases, neurological disorders, oncological disorders, ophthalmic diseases and others), phase of development (discovery, preclinical and clinical), approach of therapy (ex vivo and in vivo), cell source (autologous and allogeneic), type of therapy (CAR-T therapy, HSC therapy, T cell therapy, Phage therapy and others), and the type of technology used. 

Chapter 14 features an insightful market forecast analysis, highlighting the likely growth of novel technologies designed for the development of ZFNs, TALENs and meganucleases based therapies till the year 2035, based on licensing deal structures and agreements that are expected to be signed in the foreseen future. In addition, we estimated the likely distribution of the current and forecasted opportunity across A] type of payment (upfront payment and milestone payment) and [B] geography (North America, Europe, and Asia Pacific and rest of the world).

Chapter 15 is an appendix that contains list of all the figures and tables in the report.

Chapter 16 is an appendix that provides the list of companies and organizations mentioned in the report.

Table Of Contents

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

2. EXECUTIVE SUMMARY

3. INTRODUCTION
3.1. Overview of Genome Editing
3.2. Evolution of Genome Editing Tools
3.3. Types of Genomes Editing Tools
3.4. Zinc-finger nucleases (ZFNs): An Overview
3.4.1. Structure of ZFNs
3.4.2. Mechanism of Action
3.5. Transcription activator-like effector nucleases (TALENs): An Overview
3.5.1. Structure of TALENs
3.5.2. Mechanism of Action
3.6. Meganucleases: An Overview
3.6.1. Structure of Meganucleases
3.6.2. Mechanism of Action
3.7. Genome Editing Tools: Advantages and Disadvantages
3.8. Future Perspectives

4. ZFNS, TALENS AND MEGANUCLEASES BASED THERAPEUTICS: MARKET LANDSCAPE
4.1. Pipeline Review: ZFNs
4.1.1. Analysis by Phase of Development
4.1.2. Analysis by Biological Target(s)
4.1.3. Analysis by Delivery Vehicle
4.1.4. Analysis by Target Indication(s)
4.1.5. Analysis by Therapeutic Area
4.1.6. Analysis by Type of Therapy
4.2. Pipeline Review: TALENS
4.2.1. Analysis by Phase of Development
4.2.2. Analysis by Target Indication(s)
4.2.3. Analysis by Type of Cells
4.2.4. Analysis by Biological Target(s) and Developer
4.2.5. Analysis by Cell / Gene Therapy
4.2.6. Analysis by Type of Therapy
4.2.7. Analysis by Gene Editing Approach
4.2.8. Analysis by Route of Administration

4.3. Pipeline Review: Meganucleases
4.3.1. Analysis by Phase of Development
4.3.2. Analysis by Gene Editing Approach
4.3.3. Analysis by Delivery Vehicle
4.3.4. Analysis by Therapeutic Area
4.3.5. Analysis by Route of Administration
4.3.6. Analysis by Type of Cells
4.3.7. Analysis by Type of Therapy
4.3.8. Analysis by Biological Target(s)

4.4. Non-CRISPR based Gene Editing Therapies: Developer Landscape
4.4.1. Analysis by Year of Establishment
4.4.2. Analysis by Location of Headquarters
4.4.3. Analysis by Geographical Region
4.4.4. Analysis by Company Size
4.4.5. Analysis by Type of Player
4.4.6. Analysis by Gene Editing Technologies

5. COMPANY PROFILES
5.1. Allogene Therapeutics
5.1.1. Company Overview
5.1.2. Drug Portfolio
5.1.3. Recent Developments and Future Outlook

5.2. Bluebird Bio
5.2.1. Company Overview
5.2.2. Financial Information
5.2.3. Drug Portfolio
5.2.4. Recent Developments and Future Outlook

5.3. Cellectis
5.3.1. Company Overview
5.3.2. Financial Information
5.3.3. Drug Portfolio
5.3.4. Recent Developments and Future Outlook

5.4. Cytovia Therapeutics
5.4.1. Company Overview
5.4.2. Drug Portfolio
5.4.3. Recent Developments and Future Outlook

5.5. Iovance Therapeutics
5.5.1. Company Overview
5.5.2. Drug Portfolio
5.5.3. Recent Developments and Future Outlook

5.6. Precision Biosciences
5.6.1. Company Overview
5.6.2. Financial Information
5.6.3. Drug Portfolio
5.6.4. Recent Developments and Future Outlook

5.7. Sangamo Therapeutics
5.7.1. Company Overview
5.7.2. Financial Information
5.7.3. Drug Portfolio
5.7.4. Recent Developments and Future Outlook

6. CLINICAL TRIALS ANALYSIS
6.1. Analysis Methodology and Key Parameters
6.2. List of Clinical Trials focused on ZFN, TALEN and Meganuclease edited Therapies
6.2.1. Analysis by Trial Status
6.2.2. Analysis by Trial Registration Year
6.2.3. Analysis by Target Disease Indication
6.2.4. Analysis by Trial Phase
6.2.5. Analysis by Study Design
6.2.6. Year-wise Trend of Completed and Recruiting Trials
6.2.7. Analysis by Phase and Patient Enrollment
6.2.8. Analysis by Type of Sponsor
6.2.9. Analysis by Patient Enrollment
6.2.10. Word Cloud Analysis: Emerging Focus Areas
6.2.11. Most Active Players: Analysis by Number of Registered Trials
6.2.12. Analysis by Trial Location

7. PUBLICATION ANALYSIS
7.1. Analysis Methodology and Key Parameters
7.2. List of Publications focused on ZFN, TALEN and Meganuclease edited Therapies
7.3. Publication Analysis: ZFNs
7.3.1. Analysis by Year of Publication
7.3.2. Analysis by Therapeutic Area
7.3.3. ZFNs: Emerging Focus Areas
7.3.4. Top Journals: Analysis by Number of Publications
7.3.5. Top Authors: Analysis by Number of Publications

7.4. Publication Analysis: TALENs
7.4.1. Analysis by Year of Publication
7.4.2. Analysis by Therapeutic Area
7.4.3. TALENs: Emerging Focus Areas
7.4.4. Top Journals: Analysis by Number of Publications
7.4.5. Top Authors: Analysis by Number of Publications

7.4. Publication Analysis: Meganucleases
7.4.1. Analysis by Year of Publication
7.4.2. Meganucleases: Emerging Focus Areas
7.4.3. Top Journals: Analysis by Number of Publications
7.4.4. Top Authors: Analysis by Number of Publications

8. ACADEMIC GRANTS ANALYSIS
8.1. Analysis Methodology and Key Parameters
8.2. List of Academic Grants focused on ZFN, TALEN and Meganuclease edited Therapies
8.3. Analysis by Year of Grant Award
8.4. Analysis by Amount Awarded
8.5. Analysis by Type of Funding Institute Center
8.6. Analysis by Activity Code
8.7. Analysis by Support Period
8.8. Prominent Program Officers: Analysis by Number of Grants
8.9. Word Cloud: Emerging Focus Areas
8.9. Analysis by Type of Grant Application
8.10. Analysis by Funding Institute Center and Support Period
8.11. Popular Recipient Organizations: Analysis by Number of Grants and Amount Awarded
8.12. Popular Type of Recipient Organization: Analysis by Number of Grants
8.14. Popular Type of Recipient Organization: Analysis by Amount Awarded
8.15. Popular NIH Departments: Analysis by Number of Grants
8.16. Analysis by Study Section
8.17. Analysis by Purpose of Grant
8.18. Analysis by Amount Awarded and Purpose of Grant
8.19. Analysis by Location of Recipients

9. PARTNERSHIPS AND COLLABORATIONS
9.1. List of Partnerships and Collaborations focused on ZFNs, TALENs and Meganucleases editing Therapies
9.2. Analysis by Year of Partnership
9.3. Analysis by Type of Partnership
9.4. Analysis by Year and Type of Partnership
9.5. Analysis by Type of Technology
9.6. Spider Web Analysis: Type of Technology and Type of Agreement
9.7. Analysis by Type of Partner
9.8. Active Partners
9.9. Analysis by Year and Type of Partner
9.10. Analysis by Target Disease Indication
9.11. Analysis by Therapeutic Area
9.12. Most Active Players: Analysis by Number of Partnerships
9.13. Licensing Deals: Analysis by Upfront and Milestone Payment
9.14. Analysis by Technology and Licensing Amount
9.15. Geographical Distribution
9.16. Intercontinental and Intracontinental Agreements

10. FUNDING AND INVESTMENT ANALYSIS
10.1. Gene Editing Therapies- Focus on ZFNs, TALENs and Meganucleases: List of Funding Instances
10.2. Analysis by Year of Funding
10.3. Analysis by Amount Invested
10.4. Analysis by Type of Funding
10.5. Analysis by Year, Type of Funding and Amount Invested
10.6. Analysis by Type of Funding and Amount Invested
10.7. Industry Players: Analysis by Type of Funding
10.8 Analysis by Geographical Regions
10.9. Most Active Players: Analysis by Number of Funding Instances
10.8. Most Active Players: Analysis by Amount Invested
10.9. Key Investors: Analysis by Number of Funding Instances
10.10. Analysis of Amount Invested by Technology
10.11. Analysis by Amount Invested by Therapeutic Area

11. PATENT ANALYSIS
11.1. Analysis Methodology and Key Parameters
11.2. Patent Analysis: ZFNs
11.2.1. Analysis by Type of Patent
11.2.2. Analysis by Publication Year
11.2.3. Analysis by Type of Patent and Publication Year
11.2.4. Analysis by Issuing Authority / Patent Offices Involved
11.2.5. Analysis by CPC Symbols
11.2.6. Word Cloud: Emerging Focus Areas
11.2.7. Analysis by Type of Organization
11.2.8. Leading Industry Players: Analysis by Number of Patents
11.2.9. Analysis by Patent Age, 2001-2021
11.2.10. ZFNs: Benchmarking Patent Analysis
11.2.11. ZFNs: Patent Valuation Analysis

11.3. Patent Analysis: TALENs
11.3.1. Analysis by Type of Patent
11.3.2. Analysis by Publication Year
11.3.3. Analysis by Type of Patent and Publication Year
11.3.4. Analysis by Issuing Authority / Patent Offices Involved
11.3.5. Analysis by CPC Symbols
11.3.6. Word Cloud Analysis: Emerging Focus Areas
11.3.7. Analysis by Type of Organization
11.3.8. Leading Industry Players: Analysis by Number of Patents
11.3.9. Analysis by Patent Age, 2001-2021
11.3.10. TALENs: Benchmarking Patent Analysis
11.3.11. TALENs: Patent Valuation Analysis

11.4. Patent Analysis: Meganucleases
11.4.1. Analysis by Type of Patent
11.4.2. Analysis by Publication Year
11.4.3. Analysis by Type of Patent and Publication Year
11.4.4. Analysis by Issuing Authority / Patent Offices Involved
11.4.5. Analysis by CPC Symbols
11.4.6. Word Cloud Analysis: Emerging Focus Areas
11.4.7. Analysis by Type of Organization
11.4.8. Leading Industry Players: Analysis by Number of Patents
11.4.9. Analysis by Patent Age, 2001-2021
11.4.10. Meganucleases: Benchmarking Patent Analysis
11.4.11. Meganucleases: Patent Valuation Analysis

12. KEY INSIGHTS ON KOLs
12.1. Analysis Methodology
12.2. Key Parameters / Assumptions
12.3. Analysis by Type of Organization
12.4. Regional Analysis
12.5. Analysis by Location of Headquarters
12.6. Analysis by Current Organization
12.7. Leading KOLs by Roots Analysis Proprietary Scores
12.8. Key Insights on KOLs

13. CASE STUDY: CRISPR / CAS BASED THERAPEUTICS
13.1. CRISPR / Cas based Therapeutics: Clinical Pipeline
13.1.1. Analysis by Therapeutic Approach (In vivo / Ex vivo)
13.1.2. Analysis by Cell Source
13.1.3. Analysis by Cell Type
13.1.4. Analysis by Target Gene
13.1.5. Analysis by Delivery Vehicle Used

13.2. CRISPR / Cas Based Therapeutics: Discovery and Preclinical Pipeline
13.2.1. Analysis by Phase of Development
13.2.2. Analysis by Therapeutic Area
13.2.3. Analysis by Technology Used
13.2.4. Analysis by Therapeutic Approach (In vivo/Ex vivo)
13.2.5. Analysis by Delivery Vehicle Used
13.2.6. Most Active Players: Analysis by Number of Drugs

13.3. CRISPR / Cas based Therapeutics: Developer Landscape
13.3.1. Analysis by Year of Establishment
13.3.2. Analysis by Company Size
13.3.3. Analysis by Geographical Location
13.3.4. Logo Landscape: Analysis by Size and Phase of Development of CRISPR based Therapeutics
13.3.5. Initiatives by Big Pharma Players

14. MARKET SIZING AND OPPORTUNITY ANALYSIS
14.1. Forecast Methodology and Key Assumptions
14.2. Gene Editing Therapeutics and Technologies, Focus on ZFNs, TALENs and Meganucleases: Information on Licensing Deals
14.3. Global ZFNs, TALENs and Meganucleases based Therapeutics and Technologies Market, 2021-2035
14.4. Global ZFNs, TALENs and Meganucleases based Therapeutics and Technologies Market, 2021-2035: Distribution by Region
14.4.1. ZFNs, TALENs and Meganucleases based Therapeutics and Technologies Market in North America, 2021-2035
14.4.2. ZFNs, TALENs and Meganucleases based Therapeutics and Technologies Market in Europe, 2021-2035
14.4.3. ZFNs, TALENs and Meganucleases based Therapeutics and Technologies Market in Asia-Pacific and rest of the world, 2021-2035

15. APPNEDIX 1: LIST OF FIGURES AND TABLES

16. APPNEDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

List Of Figures

Figure 3.1 Evolution of Genome Editing Tools
Figure 3.2 Types of Genomes Editing Tools
Figure 3.3 Structure of ZFNs
Figure 3.4 Mechanism of Action of ZFNs
Figure 3.5 Structure of TALENs
Figure 3.6 Mechanism of Action of TALENs
Figure 3.7 Structure of Meganucleases
Figure 3.8 Mechanism of Action of Meganucleases
Figure 3.9 Genome Editing Tools: Advantages and Disadvantages
Figure 3.10 Future Perspectives
Figure 4.1 ZFNs based Therapeutics: Distribution by Phase of Development
Figure 4.2 ZFNs based Therapeutics: Distribution by Biological Target(s)
Figure 4.3 ZFNs based Therapeutics: Distribution by Delivery Vehicle
Figure 4.4 ZFNs based Therapeutics: Distribution by Target Indication(s)
Figure 4.5 ZFNs based Therapeutics: Distribution by Therapeutic Area
Figure 4.6 ZFNs based Therapeutics: Distribution by Type of Therapy
Figure 4.7 TALENs based Therapeutics: Distribution by Phase of Development
Figure 4.8 TALENs based Therapeutics: Distribution by Target Indication(s)
Figure 4.9 TALENs based Therapeutics: Distribution by Type of Cells
Figure 4.10 TALENs based Therapeutics: Distribution by Biological Target(s) and Developer
Figure 4.11 TALENs based Therapeutics: Distribution by Cell / Gene Therapy
Figure 4.12 TALENs based Therapeutics: Distribution by Type of Therapy
Figure 4.13 TALENs based Therapeutics: Distribution by Gene Editing Approach
Figure 4.14 TALENs based Therapeutics: Distribution by Route of Administration
Figure 4.15 Meganucleases based Therapeutics: Distribution by Phase of Development
Figure 4.16 Meganucleases based Therapeutics: Distribution by Gene Editing Approach
Figure 4.17 Meganucleases based Therapeutics: Distribution by Delivery Vehicle
Figure 4.18 Meganucleases based Therapeutics: Distribution by Therapeutic Area
Figure 4.19 Meganucleases based Therapeutics: Distribution by Route of Administration
Figure 4.20 Meganucleases based Therapeutics: Distribution by Type of Cells
Figure 4.21 Meganucleases based Therapeutics: Distribution by Type of Therapy
Figure 4.22 Meganucleases based Therapeutics: Distribution by Biological Target(s)
Figure 4.23 Non-CRISPR based Gene Editing Therapies: Distribution of Developers by Year of Establishment
Figure 4.24 Non-CRISPR based Gene Editing Therapies: Distribution of Developers by Location of Headquarters
Figure 4.25 Non-CRISPR based Gene Editing Therapies: Distribution of Developers by Geographical Region
Figure 4.26 Non-CRISPR based Gene Editing Therapies: Distribution of Developers by Company Size
Figure 4.27 Non-CRISPR based Gene Editing Therapies: Distribution of Developers by Type of Player
Figure 4.28 Non-CRISPR based Gene Editing Therapies: Distribution of Developers by Gene Editing Technologies
Figure 5.1 Bluebird Bio: Financial Information
Figure 5.2 Cellectis: Financial Information
Figure 5.3 Precision Biosciences: Financial Information
Figure 5.4 Sangamo Therapeutics: Financial Information
Figure 6.1 Clinical Trials Analysis: Distribution by Trial Status
Figure 6.2 Clinical Trials Analysis: Distribution by Trial Registration Year
Figure 6.3 Clinical Trials Analysis: Distribution by Target Disease Indication
Figure 6.4 Clinical Trials Analysis: Distribution by Trial Phase
Figure 6.5 Clinical Trials Analysis: Distribution by Study Design
Figure 6.6 Clinical Trials Analysis: Year-wise Trend of Completed and Recruiting Trials
Figure 6.7 Clinical Trials Analysis: Distribution by Phase of Development and Patient Enrollment
Figure 6.8 Clinical Trials Analysis: Distribution by Type of Sponsor
Figure 6.9 Clinical Trials Analysis: Distribution by Patient Enrollment
Figure 6.10 Clinical Trials Analysis: Distribution by Emerging Focus Areas
Figure 6.11 Most Active Players: Distribution by Number of Registered Trials
Figure 6.12 Clinical Trial Analysis: Distribution by Trial Location
Figure 7.1 Publication Analysis for ZFNs: Distribution by Year of Publication
Figure 7.2 Publication Analysis for ZFNs: Distribution by Therapeutic Area
Figure 7.3 Publication Analysis for ZFNs: Emerging Focus Areas
Figure 7.4 Top Journals: Distribution by Number of Publications for ZFNs
Figure 7.5 Top Authors: Distribution by Number of Publications for ZFNs
Figure 7.6 Publication Analysis for TALENs: Distribution by Year of Publication
Figure 7.7 Publication Analysis for TALENs: Distribution by Therapeutic Area
Figure 7.8 Publication Analysis for TALENs: Emerging Focus Areas
Figure 7.9 Top Journals: Distribution by Number of Publications for TALENs
Figure 7.10 Top Authors: Distribution by Number of Publications for TALENs
Figure 7.11 Publication Analysis for Meganucleases: Distribution by Year of Publication
Figure 7.12 Publication Analysis for Meganucleases: Emerging Focus Areas
Figure 7.13 Top Journals: Distribution by Number of Publications for Meganucleases
Figure 7.14 Top Authors: Distribution by Number of Publications for Meganucleases
Figure 8.1 Academic Grants Analysis: Distribution by Year of Grant Awarded
Figure 8.2 Academic Grants Analysis: Distribution by Amount Awarded
Figure 8.3 Academic Grants Analysis: Distribution by Type of Funding Institute Center
Figure 8.4 Academic Grants Analysis: Distribution by Activity Code
Figure 8.5 Academic Grants Analysis: Distribution by Support Period
Figure 8.6 Prominent Program Officers: Distribution by Number of Grants
Figure 8.7 Word Cloud: Emerging Focus Areas
Figure 8.8 Academic Grants Analysis: Distribution by Type of Grant Application
Figure 8.9 Academic Grants Analysis: Distribution by Funding Institute Center and Support Period
Figure 8.10 Popular Recipient Organizations: Distribution by Number of Grants and Amount Awarded
Figure 8.11 Popular Type of Recipient Organization: Distribution by Number of Grants
Figure 8.12 Popular Type of Recipient Organization: Distribution by Amount Awarded
Figure 8.13 Popular NIH Departments: Distribution by Number of Grants
Figure 8.14 Academic Grants Analysis: Distribution by Study Section
Figure 8.15 Academic Grants Analysis: Distribution by Purpose of Grant
Figure 8.16 Academic Grants Analysis: Distribution by Amount Awarded and Purpose of Grant
Figure 8.17 Academic Grants Analysis: Distribution by Location of Recipients
Figure 9.1 Partnerships and Collaborations: Distribution by Year of Partnership
Figure 9.2 Partnerships and Collaborations: Distribution by Year and Type of Partnership
Figure 9.3 Partnerships and Collaborations: Distribution by Type of Technology
Figure 9.4 Spider Web Analysis: Distribution by Type of Technology and Type of Agreement
Figure 9.5 Partnerships and Collaborations: Distribution by Type of Partner
Figure 9.6 Active Partners: Distribution by Number of Partnerships
Figure 9.7 Partnerships and Collaborations: Distribution by Year and Type of Partner
Figure 9.8 Partnerships and Collaborations: Distribution by Target Disease Indication
Figure 9.9 Partnerships and Collaborations: Distribution by Therapeutic Area
Figure 9.10 Most Active Players: Distribution by Number of Partnerships
Figure 9.11 Licensing Deals: Distribution by Upfront and Milestone Payment
Figure 9.12 Partnerships and Collaborations: Distribution by Technology and Licensing Amount
Figure 9.13 Partnerships and Collaborations: Geographical Distribution
Figure 9.14 Intercontinental and Intracontinental Agreements
Figure 10.1 Funding and Investments: Distribution by Year of Funding
Figure 10.2 Funding and Investments: Distribution by Amount Invested
Figure 10.3 Funding and Investments: Distribution by Type of Funding
Figure 10.4 Funding and Investments: Distribution by Year, Type of Funding and Amount Invested
Figure 10.5 Funding and Investments: Distribution by Type of Funding and Amount Invested
Figure 10.6 Industry Players: Distribution by Type of Funding
Figure 10.7 Funding and Investments: Distribution by Geographical Regions
Figure 10.8 Most Active Players: Distribution by Number of Funding Instances
Figure 10.9 Most Active Players: Distribution by Amount Invested
Figure 10.10 Key Investors: Distribution by Number of Funding Instances
Figure 10.11 Funding and Investments: Distribution of Amount Invested by Technology
Figure 10.12 Funding and Investments: Distribution of Amount Invested by Therapeutic Area
Figure 11.1 Patent Analysis for ZFNs: Distribution by Type of Patent
Figure 11.2 Patent Analysis for ZFNs: Distribution by Publication Year
Figure 11.3 Patent Analysis for ZFNs: Distribution by Type of Patent and Publication Year
Figure 11.4 Patent Analysis for ZFNs: Distribution by Issuing Authority / Patent Offices Involved
Figure 11.5 Patent Analysis for ZFNs: Distribution by CPC Symbols
Figure 11.6 Word Cloud Emerging Focus Areas
Figure 11.7 Patent Analysis for ZFNs: Distribution by Type of Organization
Figure 11.8 Leading Industry Players of ZFNs: Distribution by Number of Patents
Figure 11.9 Patent Analysis for ZFNs: Distribution by Patent Age, 2001-2021
Figure 11.10 Patent Analysis for ZFNs: Benchmarking Patent Analysis
Figure 11.11 Patent Analysis for ZFNs: Patent Valuation Analysis
Figure 11.12 Patent Analysis for TALENs: Distribution by Type of Patent
Figure 11.13 Patent Analysis for TALENs: Distribution by Publication Year
Figure 11.14 Patent Analysis for TALENs: Distribution by Type of Patent and Publication Year
Figure 11.15 Patent Analysis for TALENs: Distribution by Issuing Authority / Patent Offices Involved
Figure 11.16 Patent Analysis for TALENs: Distribution by CPC Symbols
Figure 11.17 Word Cloud: Emerging Focus Areas
Figure 11.18 Patent Analysis for TALENs: Distribution by Type of Organization
Figure 11.19 Leading Industry Players of TALENs: Distribution by Number of Patents
Figure 11.20 Patent Analysis for TALENs: Distribution by Patent Age, 2001-2021
Figure 11.21 Patent Analysis for TALENs: Benchmarking Patent Analysis
Figure 11.22 Patent Analysis for TALENs: Patent Valuation Analysis
Figure 11.23 Patent Analysis for Meganucleases: Distribution by Type of Patent
Figure 11.24 Patent Analysis for Meganucleases: Distribution by Publication Year
Figure 11.25 Patent Analysis for Meganucleases: Distribution by Type of Patent and Publication Year
Figure 11.26 Patent Analysis for Meganucleases: Distribution by Issuing Authority / Patent Offices Involved
Figure 11.27 Patent Analysis for Meganucleases: Distribution by CPC Symbols
Figure 11.28 Word Cloud: Emerging Focus Areas
Figure 11.29 Patent Analysis for Meganucleases: Distribution by Type of Organization
Figure 11.30 Leading Industry Players of Meganucleases: Distribution by Number of Patents
Figure 11.31 Patent Analysis for Meganucleases: Distribution by Patent Age, 2001-2021
Figure 11.32 Patent Analysis for Meganucleases: Benchmarking Patent Analysis
Figure 11.33 Patent Analysis for Meganucleases: Patent Valuation Analysis
Figure 12.1 Key Insights on KOLs: Distribution by Type of Organization
Figure 12.2 Key Insights on KOLs: Distribution by Region
Figure 12.3 Key Insights on KOLs: Distribution by Location of Headquarters
Figure 12.4 Key Insights on KOLs: Distribution by Current Organization
Figure 12.5 Leading KOLs by Roots Analysis Proprietary Scores
Figure 12.6 Key Insights on KOLs
Figure 13.1 CRISPR / Cas based Therapeutics Clinical Pipeline: Distribution by Therapeutic Approach (In vivo / Ex vivo)
Figure 13.2 CRISPR / Cas based Therapeutics Clinical Pipeline: Distribution by Cell Source
Figure 13.3 CRISPR / Cas based Therapeutics Clinical Pipeline: Distribution by Cell Type
Figure 13.4 CRISPR / Cas based Therapeutics Clinical Pipeline: Distribution by Cell Source and Cell Type
Figure 13.5 CRISPR / Cas based Therapeutics Clinical Pipeline: Distribution by Target Gene
Figure 13.6 CRISPR / Cas based Therapeutics Clinical Pipeline: Distribution by Delivery Vehicle Used and Therapeutic Approach
Figure 13.7 CRISPR / Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Phase of Development
Figure 13.8 CRISPR / Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Therapeutic Area
Figure 13.9 CRISPR / Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Technology Used
Figure 13.10 CRISPR / Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Therapeutic Approach (In vivo / Ex vivo)
Figure 13.11 CRISPR / Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Delivery Vehicle Used
Figure 13.12 Most Active Players: Distribution by Number of Drugs
Figure 13.13 CRISPR based Therapeutics Developers: Distribution by Year of Establishment
Figure 13.14 CRISPR / Cas based Therapeutics Developers: Distribution by Company Size
Figure 13.15 CRISPR / Cas based Therapeutics Developers: Distribution by Geographical Location
Figure 13.16 Logo Landscape of CRISPR / Cas based Therapeutics Developers: Distribution by Size and Phase of Development
Figure 14.1 Licensing Agreements: Distribution of Financial Components
Figure 14.2 Technology Licensing Deals: Payment Structure
Figure 14.3 Gene Editing Technologies: Global Market Opportunity, 2021-2035
Figure 14.4 Gene Editing Technologies: Distribution by Type of Payment
Figure 14.5 Gene Editing Technologies: Distribution by Geography, 2021 and 2035
Figure 14.6 Gene Editing Technologies: Market Opportunity in North America, 2021-2035
Figure 14.7 Gene Editing Technologies: Market by Upfront Payment in North America, 2021-2035
Figure 14.8 Gene Editing Technologies: Market by Milestone Payment in North America, 2021-2035
Figure 14.9 Gene Editing Technologies: Market Opportunity in Europe, 2021-2035
Figure 14.10 Gene Editing Technologies: Market by Upfront Payment in Europe, 2021-2035
Figure 14.11 Gene Editing Technologies: Market by Milestone Payment in Europe, 2021-2035
Figure 14.12 Gene Editing Technologies: Market Opportunity in Asia-Pacific and RoW, 2021-2035
Figure 14.13 Gene Editing Technologies: Market by Upfront Payment in Asia-Pacific and RoW, 2021-2035
Figure 14.14 Gene Editing Technologies: Market by Milestone Payment in Asia-Pacific and RoW, 2021-2035

List Of Tables

Table 4.1 ZFN based Therapies: Clinical Pipeline
Table 4.2 TALEN based Therapies: Clinical Pipeline
Table 4.3 Meganucleases based Therapies: Clinical Pipeline
Table 4.4 Gene Editing Therapies: List of Developers
Table 4.5 Gene Editing Technologies: List of Developers
Table 6.1 List of Clinical Trials, till 2021
Table 7.1 List of Publications, 2016-2021
Table 8.1 List of Academic Grants, till 2021
Table 9.1 List of Partnerships and Collaborations, till 2021
Table 10.1 List of Funding Instances, till 2021
Table 11.1 List of Patents, 2013-2021
Table 12.1 List of KOLs
Table 13.1 CRISPR / Cas based Therapeutics: Clinical Pipeline

List Of Companies

The following companies / institutes / government bodies and organizations have been mentioned in this report.

  1. Adage Capital Management
  2. Agent Capital
  3. Alexandria Venture Investments
  4. Allogene Therapeutics
  5. Amgen Ventures
  6. ArrowMark Partners
  7. Baxter Ventures
  8. BellCo Capital
  9. Biogen
  10. Bioverativ (part of Sanofi)
  11. bluebird bio
  12. Brace Pharma Capital
  13. California Institute for Regenerative Medicine
  14. Cellectis
  15. City of Hope National Medical Center
  16. Cormorant Asset Management
  17. Cowen Healthcare Investments
  18. Cytovia Therapeutics
  19. Deerfield Management
  20. Ditch Plains Capital Management
  21. Dow AgroSciences
  22. DSAM Partners
  23. DUMAC
  24. Edmond de Rothschild
  25. Eli Lilly and Company
  26. Eurazeo
  27. Fidelity Management and Research Company
  28. Fox Chase Cancer Center
  29. F-Prime Capital
  30. Franklin Templeton Investments
  31. Fred Hutchinson Cancer Research Center
  32. Gilead Sciences
  33. Huazhong University of Science and Technology
  34. Iovance Therapeutics
  35. Iowa State University
  36. J.P. Morgan
  37. Jacobi Medical Center
  38. Jennison Associates
  39. King's College Hospital
  40. Kite Pharma (a Gilead Company)
  41. Laurion Capital Management
  42. Lilly Asia Ventures
  43. Massachusetts General Hospital
  44. Mayo Clinic
  45. MD Anderson Cancer Center
  46. Medical College of Wisconsin
  47. Mogrify 
  48. National Heart, Lung, and Blood Institute
  49. National Human Genome Research Institute
  50. National Institute of Allergy and Infectious Diseases
  51. National Institutes of Health
  52. Novartis
  53. Novo Nordisk
  54. OCV
  55. ODYSSEE Venture
  56. Osage University Partners
  57. Perceptive Advisors
  58. Pfizer
  59. Pontifax AgTech
  60. Precision BioSciences
  61. RA Capital Management
  62. Ridgeback Capital Management
  63. Sangamo Therapeutics
  64. Servier
  65. Sigma-Aldrich
  66. SpringWorks Therapeutics
  67. Stanford University
  68. Sun Yat-sen University
  69. Surveyor Capital
  70. SVB Leerink
  71. T. Rowe Price Associates
  72. Takeda
  73. The Jackson Laboratory
  74. The Longevity Fund
  75. The University of Utah
  76. UMass Chan Medical School
  77. University of California
  78. University of Cincinnati
  79. University of Michigan
  80. University of Minnesota
  81. University of Pennsylvania
  82. University of Southern California
  83. University of Washington
  84. UT Southwestern Medical Center
  85. venBio
  86. venBio Select Advisor
  87. Vida Ventures
  88. Vivo Capital
  89. Weill Cornell Medicine
  90. Wyss Institute for Biologically Inspired Engineering
  91. Yale University
  92. Yeda Research & Development

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