CRISPR-based Therapeutics

Chapter Outlines

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

Chapter 3 provides a general introduction to CRISPR/Cas system. In addition, we have briefly described the components of CRISPR/Cas system, its mechanism of action and vehicles to deliver CRISPR/Cas components in to the target cells. The chapter lays emphasis on the applications of CRISPR technology. It also includes a discussion on the challenges associated with the use of CRISPR based therapeutics.

Chapter 4 presents a detailed assessment of the current market landscape of CRISPR based therapeutics, along with information on type of therapy (CAR-T therapy, HSC therapy, T cell therapy, Phage therapy and others), approach of therapy (in vivo and ex vivo), cell source (autologous and allogeneic), phase of development (discovery, preclinical and clinical), type of delivery vehicle used (viral vector, electroporation, LNPs, bacteriophage and others), target disease indication and therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscular diseases, neurological disorders, oncological disorders, ophthalmic diseases and others). In addition, it provides an overview of the CRISPR based therapeutics developer landscape, highlighting the players that are active in this domain. It includes information on their year of establishment, company size (in terms of number of employees) and location of headquarters of the drug developers. We have presented a logo landscape, highlighting the distribution of the drug developers based on company size and location of headquarters. Further, it presents an analysis on the initiatives of the big pharma companies in this domain.

Chapter 5 includes profiles of the key players engaged in the development of CRISPR based therapeutics (shortlisted based on strength of product portfolio). Each profile features a brief overview of the company, its financial information (if available), brief details of gene editing technology, therapeutic pipeline, recent developments and an informed future outlook.

Chapter 6 provides an in-depth analysis of the patents filed / granted for CRISPR technology since 2015. The analysis also highlights the key trends associated with these patents, including type of patent (granted patent, patent application and others), publication year, application year, geographical location / patent jurisdiction (North America, Europe, Asia-Pacific and Rest of the World), IPCR symbols, key inventors and leading industry / non-industry players. In addition, it includes a detailed patent benchmarking analysis of leading players and patent valuation analysis, which evaluates the qualitative and quantitative aspects of these patents.

Chapter 7 features a detailed analysis of the partnerships and collaborations that have been inked in this domain since 2014, covering research and licensing agreements, R&D agreements, licensing agreements, licensing and manufacturing agreement, product development and manufacturing agreements, joint ventures and other types of partnership deals. The chapter includes analysis based on year of partnership, type of partnership model, purpose of licensing deal, and most active player(s) (in terms of number of partnerships inked). In addition, the chapter features a discussion on the surrogate licensing practice in the CRISPR based therapeutics market.

Chapter 8 provides an analysis of the investments made since 2014 at various stages of development of companies engaged in this domain, based on the year of investment, number of funding instances, amount invested and type of funding, highlighting most active players (in terms of number of funding instances and amount raised) and most active investors (in terms of number of funding instances).

Chapter 9 presents an analysis of the start-ups (established after 2012 and having less than 200 employees) engaged in the development of CRISPR based therapeutics based on the parameters, such as number of candidates in discovery, preclinical and clinical phase of development, target therapeutic area, amount raised through funding, number of investors, type of funding, number of deals signed, and number of patents filed. 

Chapter 10 features an elaborate discussion on the future market potential of various CRISPR based therapeutics. The chapter provides insights on the likely distribution of the current and forecasted opportunity across [A] target therapeutic area (hematological disorders, oncological disorders, ophthalmic diseases, infectious diseases and others) [B] approach of therapy (ex vivo and in vivo), [C] type of therapy (CAR-T cell therapy, HSC therapy, T cell therapy, and TIL) and [D] key geographical regions (North America, Europe and Asia-Pacific). 

Chapter 11 is a summary of the overall report, highlighting the key facts and figures related to the research and analysis presented in the previous chapters.

Chapter 12 is a collection of interview transcripts of discussions held with representatives of renowned organizations engaged in the CRISPR technology domain. In this chapter, we have presented the insights on our conversation with Harrison Wong (Public Relations, Burns McClellan, for eGenesis).

Chapter 13 is an appendix that contains tabulated data and numbers for all the figures provided in the report.

Chapter 14 is an appendix, which consists 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. Chapter Overview
3.2. CRISPR/Cas System
3.2.1. Mechanism of Action
3.2.2. Innovations in CRISPR/Cas based Gene Editing
3.4. CRISPR/Cas Component Delivery Vehicles
3.4.1. Physical Delivery
3.4.2. Viral Delivery
3.4.3. Non-Viral Delivery
3.5. Applications of the CRISPR/Cas System
3.6. CRISPR/Cas based Therapeutics
3.7. Challenges and Future Prospects

4. MARKET OVERVIEW
4.1. Chapter Overview
4.2. CRISPR/Cas based Therapeutics: Clinical Pipeline
4.2.1. Analysis by Therapeutic Approach (In vivo / Ex vivo)
4.2.2. Analysis by Cell Source
4.2.3. Analysis by Cell Type
4.2.4. Analysis by Target Gene
4.2.5. Analysis by Delivery Vehicle Used

4.3. CRISPR/Cas based Therapeutics: Registered Clinical Trials
4.3.1. Analysis by Trial Phase
4.3.2. Analysis by Therapeutic Area
4.3.3. CRISPR/Cas based Therapeutics Clinical Trials: Analysis by Target Gene and Therapeutic Area
4.3.4. Analysis by Gene Editing Approach
4.3.5. Most Active Players: Analysis by Number of Trials

4.4. CRISPR Based Therapeutics: Discovery and Preclinical Pipeline
4.4.1. Analysis by Phase of Development
4.4.2. Analysis by Therapeutic Area
4.4.3. Analysis by Technology Used
4.4.4. Analysis by Therapeutic Approach (In vivo/Ex vivo)
4.4.5. Analysis by Delivery Vehicle Used
4.4.6. Most Active Players: Analysis by Number of Drugs

4.5. CRISPR/Cas based Therapeutics: Developer Landscape
4.5.1. Analysis by Year of Establishment
4.5.2. Analysis by Company Size
4.5.3. Analysis by Geographical Location
4.5.4. Logo Landscape: Analysis by Size and Phase of Development of CRISPR based Therapeutics
4.5.5. Initiatives of Big Pharma Players

5. COMPANY PROFILES
5.1. Chapter Overview
5.2. CRISPR Therapeutics
5.2.1. Company Overview
5.2.2. Technology Overview
5.2.3. CRISPR Therapeutics: Product Pipeline
5.2.4. Recent Developments and Future Outlook

5.3. Editas Medicine
5.3.1. Company Overview
5.3.2. Technology Overview
5.3.3. Editas Medicine: Product Pipeline
5.3.4. Recent Developments and Future Outlook

5.4. Intellia Therapeutics
5.4.1. Company Overview
5.4.2. Technology Overview
5.4.3. Intellia Therapeutics: Product Pipeline
5.4.4. Recent Developments and Future Outlook

5.5. Excision BioTherapeutics
5.5.1. Company Overview
5.5.2. Technology Overview
5.5.3. Excision BioTherapeutics: Product Pipeline
5.5.4. Recent Developments and Future Outlook

5.6. Locus Biosciences
5.6.1. Company Overview
5.6.2. Technology Overview
5.6.3. Locus Biosciences: Product Pipeline
5.6.4. Recent Developments and Future Outlook

5.7. Graphite Bio
5.7.1. Company Overview
5.7.2. Technology Overview
5.7.3. Graphite Bio: Product Pipeline
5.7.4. Recent Developments and Future Outlook

5.8. Emendo Biotherapeutics (A Subsidiary of AnGes)
5.8.1. Company Overview
5.8.2. Technology Overview
5.8.3. Emendo Biotherapeutics: Product Pipeline
5.8.4. Recent Developments and Future Outlook

5.9. Beam Therapeutics
5.9.1. Company Overview
5.9.2. Technology Overview
5.9.3. Beam Therapeutics: Product Pipeline
5.9.4. Recent Developments and Future Outlook

5.10. Modalis Therapeutics
5.10.1. Company Overview
5.10.2. Technology Overview
5.10.3. Modalis Therapeutics: Product Pipeline
5.10.4. Recent Developments and Future Outlook

5.11. Refuge Biotechnologies
5.11.1. Company Overview
5.11.2. Technology Overview
5.11.3. Refuge Biotechnologies: Product Pipeline
5.11.4. Recent Developments and Future Outlook

6. PATENT ANALYSIS
6.1. Chapter Overview
6.2. Scope and Methodology
6.3. CRISPR/Cas Gene Editing Technology: Patent Analysis
6.3.1. Analysis by Publication Year and Patent Type, Pre-2015-2020
6.3.2. Analysis by Patent Application Area
6.3.3. Analysis by Patent Issuing Authority
6.3.4. Analysis by Geographical Location
6.3.5. Analysis by IPCR Symbols
6.3.6. Analysis by Type of Organization
6.3.7. Leading Players: Analysis by Number of Patents

6.4. CRISPR/Cas Gene Editing Technology: Patent Benchmarking
6.4.1. Analysis by Patent Characteristics

6.5. CRISPR/Cas Gene Editing Technology: Patent Valuation Analysis
6.6. Popular Patents: Analysis by Number of Citations

7. PARTNERSHIPS AND COLLABORATIONS
7.1. Chapter Overview
7.2. Partnership Models
7.3. CRISPR Licensing Landscape: Surrogate Licensing
7.4. CRISPR/Cas based Therapeutics: Recent Partnerships
7.4.1. Analysis by Year of Partnership
7.4.2. Analysis by Type of Partnership
7.4.3. Analysis by Purpose of Licensing
7.4.4. Most Active Players: Analysis by Number of Partnerships

8. FUNDING AND INVESTMENT ANALYSIS
8.1. Chapter Overview
8.2. Types of Funding Instances
8.3. CRISPR/Cas based Therapeutics and Affiliated Technologies: Recent Funding Instances
8.3.1. Analysis by Year of Investment
8.3.2. Analysis by Amount Invested
8.3.3. Analysis by Type of Funding
8.3.4. Most Active Players: Analysis by Number of Funding Instances
8.3.5. Most Active Investors: Analysis by Number of Funding Instances
8.4. Concluding Remarks

9. START-UP HEALTH INDEXING
9.1. Chapter Overview
9.2. Start-ups Focused on CRISPR/Cas based Therapeutics
9.3.1. Analysis by Location of Headquarters

9.4 Benchmarking of Start-ups
9.4.1. Analysis by Portfolio Strength
9.4.2. Analysis by Pipeline Maturity
9.4.3. Analysis by Therapeutic Diversity
9.4.4. Analysis by Capital Accrued through Investments
9.4.5. Analysis by Number of Investors
9.4.6. Analysis of Partnership Activity
9.4.7. Analysis of Patent Portfolio
9.4.8. Start-ups Health Indexing: Roots Analysis Perspective

10. MARKET SIZING AND OPPORTUNITY ANALYSIS
10.1. Chapter Overview
10.2. Scope and Limitations
10.3. Key Assumptions and Forecast Methodology
10.4. Overall CRISPR/Cas based Therapeutics Market, 2021-2030
10.5. CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Type of Therapy
10.6. CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Therapeutic Approach
10.7. CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Therapeutic Area
10.8. CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Geographical Region
10.9. Product-wise Sales Forecast
10.9.1. EDIT-101 (Editas Medicine)
10.9.1.1. Target Patient Population
10.9.1.2. Sales Forecast (USD Million)

10.9.2. OTQ923 / HIX763 (Intellia Therapeutics / Novartis)
10.9.2.1. Target Patient Population
10.9.2.2. Sales Forecast (USD Million)

10.9.3. CTX001 for Sickle Cell (CRISPR Therapeutics)
10.9.3.1. Target Patient Population
10.9.3.2. Sales Forecast (USD Million)

10.9.4. CTX001 for Transfusion-Dependent Beta Thalassemia (CRISPR Therapeutics)
10.9.4.1. Target Patient Population
10.9.4.2. Sales Forecast (USD Million)

10.9.5. Unnamed Drug for Metastatic Gastrointestinal Epithelial Cancer (Intima Bioscience)
10.9.5.1. Target Patient Population
10.9.5.2. Sales Forecast (USD Million)

10.9.6. BD111 (Shanghai BD Gene Therapeutics)
10.9.6.1. Target Patient Population
10.9.6.2. Sales Forecast (USD Million)

10.9.7. Unnamed Drug for Beta Thalassemia Major (Shanghai Bioray Laboratories)
10.9.7.1. Target Patient Population
10.9.7.2. Sales Forecast (USD Million)

10.9.8. CD19-UCART (Shanghai Bioray Laboratories)
10.9.8.1. Target Patient Population
10.9.8.2. Sales Forecast (USD Million)

10.9.9. Unnamed Drug for Advanced Non-Small Cell Lung Cancer (Guangzhou Anjie Biomedical Technology)
10.9.9.1. Target Patient Population
10.9.9.2. Sales Forecast (USD Million)

10.9.10. Unnamed Drug for Advanced Prostate Cancer (Guangzhou Anjie Biomedical Technology)
10.9.10.1. Target Patient Population
10.9.10.2. Sales Forecast (USD Million)

11. EXECUTIVE INSIGHTS
11.1 Chapter Overview
11.2 eGenesis
11.2.1. Company Snapshot
11.2.2. Interview Transcript: Harisson Wong (Public Relations, Burns McClellan, for eGenesis

12. CONCLUDING REMARKS

13. APPENDIX 1: TABULATED DATA

14. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

List Of Figures

Figure 3.1 Structure of CRISPR/Cas
Figure 3.2 DNA Repair Mechanism Used in CRISPR/Cas System
Figure 3.3 CRISPR/Cas System: Mechanism of Action
Figure 3.4 Applications of CRISPR/Cas System
Figure 4.1 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Therapeutic Approach (In vivo / Ex vivo)
Figure 4.2 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Cell Source
Figure 4.3 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Cell Type
Figure 4.4 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Cell Source and Cell Type
Figure 4.5 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Target Gene
Figure 4.6 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Delivery Vehicle Used and Therapeutic Approach
Figure 4.7 CRISPR/Cas based Therapeutics Clinical Trials: Distribution by Trial Phase
Figure 4.8 CRISPR/Cas based Therapeutics Clinical Trials: Distribution by Therapeutic Area
Figure 4.9 CRISPR/Cas based Therapeutics Clinical Trials: Distribution by Target Gene and Therapeutic Area
Figure 4.10 CRISPR/Cas based Therapeutics Clinical Trials: Distribution by Gene Editing Approach
Figure 4.11 Most Active Players: Distribution by Number of trials
Figure 4.12 CRISPR/Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Phase of Development
Figure 4.13 CRISPR/Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Therapeutic Area
Figure 4.14 CRISPR/Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Technology Used
Figure 4.15 CRISPR/Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Therapeutic Approach (In vivo/Ex vivo)
Figure 4.16 CRISPR/Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Delivery Vehicle Used
Figure 4.17 Most Active Players: Distribution by Number of Drugs
Figure 4.18 CRISPR based Therapeutics Developers: Distribution by Year of Establishment
Figure 4.19 CRISPR/Cas based Therapeutics Developers: Distribution by Company Size
Figure 4.20 CRISPR/Cas based Therapeutics Developers: Distribution by Geographical Location
Figure 4.21 CRISPR/Cas based Therapeutics Developers: Logo Landscape
Figure 6.1 Patent Analysis: Distribution by Patent Type
Figure 6.2 Patent Analysis: Cumulative Distribution by Publication Year, Pre 2015-2020
Figure 6.3 Patent Analysis: Distribution by Publication Year and Patent Type, Pre-2015-2020
Figure 6.4 Patent Analysis: Distribution by Patent Application Area
Figure 6.5 Patent Analysis: Distribution by Patent Issuing Authority
Figure 6.6 Patent Analysis: Distribution by Geographical Location
Figure 6.7 Patent Analysis: Distribution by IPCR Symbols
Figure 6.8 Patent Analysis: Cumulative Year-wise Distribution by Type of Organization
Figure 6.9 Patent Analysis: Leading Industry Players
Figure 6.10 Patent Analysis: Leading Non-Industry Players
Figure 6.11 Patent Analysis: Patent Assignees
Figure 6.12 Patent Analysis (Leading Players): Benchmarking by Patent Characteristics (IPCR Symbols)
Figure 6.13 Patent Analysis: Distribution of Patents by Age (2001-2020)
Figure 6.14 CRISPR/Cas Gene Editing Technology: Patent Valuation
Figure 7.1 CRISPR Licensing Landscape: Surrogate Licensing
Figure 7.2 Partnerships and Collaborations: Cumulative Year-wise Trend, 2015-2021 (till February)
Figure 7.3 Partnerships and Collaborations: Distribution by Type of Partnership
Figure 7.4 Partnerships and Collaborations: Year-wise Trend by Type of Partnership, 2015-2021 (till February)
Figure 7.5 Licensing Agreements: Distribution by Type of Licensing
Figure 7.6 Licensing Agreements: Distribution by Purpose of Licensing
Figure 7.7 Most Active Players: Distribution by Number of Partnerships
Figure 8.1 CRISPR/Cas based Therapeutics and Affiliated Technologies: List of Funding Instances, 2014-2021 (till February)
Figure 8.2 Funding and Investments: Cumulative Amount Invested by Year, 2014-2021 (till February) (USD Million)
Figure 8.3 Funding and Investments: Distribution by Type of Funding
Figure 8.4 Funding and Investments: Distribution of Amount Invested by Type of Funding (USD Million)
Figure 8.5 Funding Instances: Year-wise Trend of Amount Invested, 2014-2021 (till February) (USD Million)
Figure 8.6 Funding and Investments: Summary of Investments, 2014-2021 (till February) (USD Million)
Figure 8.7 Most Active Players: Distribution by Number of Funding Instances
Figure 8.8 Most Active Players: Distribution by Amount Raised (USD Million)
Figure 8.9 Most Active Investors: Distribution by Number of Funding Instances
Figure 8.10 Summary of Investments
Figure 9.1 Start-ups focused on CRISPR/Cas based Therapeutics: Distribution by Location of Headquarters
Figure 9.2 Benchmarking of Start-ups: Distribution by Portfolio Strength
Figure 9.3 Benchmarking of Start-ups: Distribution by Pipeline Maturity
Figure 9.4 Benchmarking of Start-ups: Mapping by Year of Establishment and Clinical Development
Figure 9.5 Benchmarking of Start-ups: Distribution by Therapeutic Diversity
Figure 9.6 Benchmarking of Start-ups: Distribution by Capital Accrued through Investments (USD Million)
Figure 9.7 Benchmarking of Start-ups: Distribution by Number of Investors
Figure 9.8 Benchmarking of Start-ups: Distribution by Partnership Activity
Figure 9.9 Benchmarking of Start-ups: Distribution by Patent Portfolio
Figure 9.10 Start-up Health Indexing: Roots Analysis Perspective
Figure 9.11 Start-up Health Indexing: Leading Companies
Figure 10.1 Global CRISPR/Cas based Therapeutics Market, 2021-2030 (USD Million)
Figure 10.2 CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Type of Therapy (USD Million)
Figure 10.3 CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Therapeutic Approach (USD Million)
Figure 10.4 CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Therapeutic Area (USD Million)
Figure 10.5 CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Geographical Region (USD Million)
Figure 10.6 EDIT-101 Target Patient Population (Till 2030)
Figure 10.7 EDIT-101 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 10.8 OTQ923 / HIX763 Target Patient Population (Till 2030)
Figure 10.9 OTQ923 / HIX763 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 10.10 CTX001 for Sickle Cell Disease Target Patient Population (Till 2030)
Figure 10.11 CTX001 for Sickle Cell Disease Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 10.12 CTX001 for Transfusion-Dependent β-Thalassemia Target Patient Population (Till 2030)
Figure 10.13 CTX001 for Transfusion-Dependent β-Thalassemia Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 10.14 Unnamed Drug for Metastatic Gastrointestinal Epithelial Cancer Target Patient Population (Till 2030)
Figure 10.15 Unnamed Drug for Metastatic Gastrointestinal Epithelial Cancer Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 10.16 BD111 Target Patient Population (Till 2030)
Figure 10.17 BD111 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 10.18 Unnamed Drug for Beta Thalassemia Major Target Patient Population (Till 2030)
Figure 10.19 Unnamed Drug for Beta Thalassemia Major Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 10.20 CD19-UCART Target Patient Population (Till 2030)
Figure 10.21 CD19-UCART Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 10.22 Unnamed Drug for Advanced Non-Small Cell Lung Cancer Target Patient Population (Till 2030)
Figure 10.23 Unnamed Drug for Advanced Non-Small Cell Lung Cancer Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 10.24 Unnamed Drug for Advanced Prostate Cancer Target Patient Population (Till 2030)
Figure 10.25 Unnamed Drug for Advanced Prostate Cancer Sales Forecast (Till 2030): Base Scenario (USD Million)

List Of Tables

Table 4.1 CRISPR/Cas based Therapeutics: Clinical Pipeline
Table 4.2 CRISPR/Cas based Therapeutics: Registered Clinical Trials
Table 4.3 CRISPR/Cas based Therapeutics: Discovery and Preclinical Pipeline
Table 4.4 CRISPR/Cas based Therapeutics: List of Developers
Table 4.5 Big Pharma Initiatives
Table 5.1 CRISPR/Cas based Therapeutics: List of Companies Profiled
Table 5.2 CRISPR Therapeutics: Company Snapshot
Table 5.3 CRISPR Therapeutics: Product Pipeline
Table 5.4 CRISPR Therapeutics: Recent Developments and Future Outlook
Table 5.5 Editas Medicine: Company Snapshot
Table 5.6 Editas Medicine: Product Pipeline
Table 5.7 Editas Medicine: Recent Developments and Future Outlook
Table 5.8 Intellia Therapeutics: Company Snapshot
Table 5.9 Intellia Therapeutics: Product Pipeline
Table 5.10 Intellia Therapeutics: Recent Developments and Future Outlook
Table 5.11 Excision BioTherapeutics: Company Snapshot
Table 5.12 Excision BioTherapeutics: Product Pipeline
Table 5.13 Excision BioTherapeutics: Recent Developments and Future Outlook
Table 5.14 Locus Biosciences: Company Snapshot
Table 5.15 Locus Biosciences: Product Pipeline
Table 5.16 Locus Biosciences: Recent Developments and Future Outlook
Table 5.17 Graphite Bio: Company Snapshot
Table 5.18 Graphite Bio: Product Pipeline
Table 5.19 Graphite Bio: Recent Developments and Future Outlook
Table 5.20 Emendo Biotherapeutics: Company Snapshot
Table 5.21 Emendo Biotherapeutics: Product Pipeline
Table 5.22 Emendo Biotherapeutics: Recent Developments and Future Outlook
Table 5.23 Beam Therapeutics: Company Snapshot
Table 5.24 Beam Therapeutics: Product Pipeline
Table 5.25 Beam Therapeutics: Recent Developments and Future Outlook
Table 5.26 Modalis Therapeutics: Company Snapshot
Table 5.27 Modalis Therapeutics: Product Pipeline
Table 5.28 Modalis Therapeutics: Recent Developments and Future Outlook
Table 5.29 Refuge Biotechnologies: Company Snapshot
Table 5.30 Refuge Biotechnologies: Product Pipeline
Table 5.31 Refuge Biotechnologies: Recent Developments and Future Outlook
Table 6.1 Patent Analysis: IPCR Class Symbol Definitions
Table 6.2 Patent Analysis: IPCR Symbol Definitions
Table 6.3 Patent Analysis: IPCR Classification Symbol Definitions
Table 6.4 Patent Analysis: Summary of Benchmarking Analysis
Table 6.5 Patent Analysis: Categorization based on Weighted Valuation Scores
Table 6.6 Patent Portfolio: List of Leading Patents (by Highest Relative Valuation)
Table 6.7 Patent Portfolio: List of Popular Patents (by Number of Citations)
Table 7.1 CRISPR/Cas based Therapeutics: List of Partnerships, 2015-2021 (till February)
Table 7.2 Most Active Players: Distribution by Number of Partnerships
Table 8.1 CRISPR/Cas based Therapeutics and Affiliated Technologies: List of Funding Instances, 2014-2021 (till February)
Table 8.2 Funding and Investments: Summary of Investments
Table 9.1 CRISPR/Cas Based Therapeutics Start-ups: Distribution by Location of Headquarters
Table 10.1 CRISPR/Cas based Therapeutics: Expected Launch Years of Advanced Stage Drug Candidates
Table 13.1 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Therapeutic Approach (In vivo / Ex vivo)
Table 13.2 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Cell Source
Table 13.3 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Cell Type
Table 13.4 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Cell Source and Cell Type
Table 13.5 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Target Gene
Table 13.6 CRISPR/Cas based Therapeutics Clinical Pipeline: Distribution by Delivery Vehicle Used and Therapeutic Approach
Table 13.7 CRISPR/Cas based Therapeutics Clinical Trials: Distribution by Phase of Development
Table 13.8 CRISPR/Cas based Therapeutics Clinical Trials: Distribution by Therapeutic Area
Table 13.9 CRISPR/Cas based Therapeutics Clinical Trials: Distribution by Gene Editing Approach
Table 13.10 Most Active Players: Distribution by Number of trials
Table 13.11 CRISPR/Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Phase of Development
Table 13.12 CRISPR/Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Therapeutic Area
Table 13.13 CRISPR/Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Technology Used
Table 13.14 CRISPR/Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Therapeutic Approach (In vivo / Ex vivo)
Table 13.15 CRISPR/Cas based Therapeutics Discovery and Preclinical Pipeline: Distribution by Delivery Vehicle Used
Table 13.16 Most Active Players: Distribution by Number of Drugs
Table 13.17 CRISPR/Cas based Therapeutics Developers: Distribution by Year of Establishment
Table 13.18 CRISPR/Cas based Therapeutics Developers: Distribution by Company Size
Table 13.19 CRISPR/Cas based Therapeutics Developers: Distribution by Geographical Location
Table 13.20 Patent Analysis: Distribution by Patent Type
Table 13.21 Patent Analysis: Cumulative Distribution by Publication Year, Pre-2015-2020
Table 13.22 Patent Analysis: Distribution by Publication Year and Patent Type, Pre-2015-2020
Table 13.23 Patent Analysis: Distribution by Patent Application Area
Table 13.24 Patent Analysis: Distribution by Patent Issuing Authority
Table 13.25 Patent Analysis: Distribution by Geographical Location
Table 13.26 Patent Analysis: Cumulative Year-wise Distribution by Type of Organization
Table 13.27 Patent Analysis: Leading Industry Players
Table 13.28 Patent Analysis: Leading Non-Industry Players
Table 13.29 Patent Analysis: Patent Assignees
Table 13.30 Patent Analysis: Distribution of Patents by Age (2001-2020)
Table 13.31 Partnerships and Collaborations: Cumulative Year-wise Trend, 2015-2021 (till February)
Table 13.32 Partnerships and Collaborations: Distribution by Type of Partnership
Table 13.33 Partnerships and Collaborations: Year-wise Trend by Type of Partnership, 2015-2021 (till February)
Table 13.34 Licensing Agreements: Distribution by Type of Licensing
Table 13.35 Licensing Agreements: Distribution by Purpose of Licensing
Table 13.36 Most Active Players: Distribution by Number of Partnerships
Table 13.37 CRISPR/Cas based Therapeutics and Affiliated Technologies: List of Funding Instances, 2014-2021 (till February)
Table 13.38 Funding and Investments: Cumulative Amount Invested by Year, 2014-2021 (till February) (USD Million)
Table 13.39 Funding and Investments: Distribution of by Type of Funding
Table 13.40 Funding and Investments: Distribution of Amount Invested by Type of Funding (USD Million)
Table 13.41 Most Active Players: Distribution by Number of Funding Instances
Table 13.42 Most Active Players: Distribution by Amount Raised (USD Million)
Table 13.43 Most Active Investors: Distribution by Number of Funding Instances
Table 13.44 Start-ups focused on CRISPR/Cas based Therapeutics: Distribution by Location of Headquarters
Table 13.45 Benchmarking of Start-ups: Distribution by Portfolio Strength
Table 13.46 Benchmarking of Start-ups: Distribution by Pipeline Drugs by Stage of Development
Table 13.47 Benchmarking of Start-ups: Distribution by Therapeutic Area
Table 13.48 Benchmarking of Start-ups: Distribution by Capital Accrued through Investments (USD Million)
Table 13.49 Benchmarking of Start-ups: Distribution by Number of Investors
Table 13.50 Benchmarking of Start-ups: Distribution by Partnership Activity
Table 13.51 Benchmarking of Start-ups: Distribution by Patent Portfolio
Table 13.52 Global CRISPR/Cas based Therapeutics Market, 2021-2030 (USD Million)
Table 13.53 CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Type of Therapy (USD Million)
Table 13.54 CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Therapeutic Approach (USD Million)
Table 13.55 CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Therapeutic Area (USD Million)
Table 13.56 CRISPR/Cas based Therapeutics Market, 2021-2030: Distribution by Geographical Region (USD Million)
Table 13.57 EDIT-101 Target Patient Population (Till 2030)
Table 13.58 EDIT-101 Sales Forecast (Till 2030) (USD Million)
Table 13.59 OTQ923 / HIX763 Target Patient Population (Till 2030)
Table 13.60 OTQ923 / HIX763 Sales Forecast (Till 2030) (USD Million)
Table 13.61 CTX001 for Sickle Cell Disease Target Patient Population (Till 2030)
Table 13.62 CTX001 for Sickle Cell Disease Sales Forecast (Till 2030) (USD Million)
Table 13.63 CTX001 for Transfusion-Dependent β-Thalassemia Target Patient Population (Till 2030)
Table 13.64 CTX001 for Transfusion-Dependent β-Thalassemia Sales Forecast (Till 2030) (USD Million)
Table 13.65 Unnamed Drug for Metastatic Gastrointestinal Epithelial Cancer Target Patient Population (Till 2030)
Table 13.66 Unnamed Drug for Metastatic Gastrointestinal Epithelial Cancer Sales Forecast (Till 2030) (USD Million)
Table 13.67 BD111 Target Patient Population (Till 2030)
Table 13.68 BD111 Sales Forecast (Till 2030) (USD Million)
Table 13.69 Unnamed Drug for Beta Thalassemia Major Target Patient Population (Till 2030)
Table 13.70 Unnamed Drug for Beta Thalassemia Major Sales Forecast (Till 2030) (USD Million)
Table 13.71 CD19-UCART Target Patient Population (Till 2030)
Table 13.72 CD19-UCART Sales Forecast (Till 2030) (USD Million)
Table 13.73 Unnamed Drug for Advanced Non-Small Cell Lung Cancer Target Patient Population (Till 2030)
Table 13.74 Unnamed Drug for Advanced Non-Small Cell Lung Cancer Sales Forecast (Till 2030) (USD Million)
Table 13.75 Unnamed Drug for Advanced Prostate Cancer Target Patient Population (Till 2030)
Table 13.76 Unnamed Drug for Advanced Prostate Cancer Sales Forecast (Till 2030) (USD Million)

List Of Companies

The following companies and organizations have been mentioned in the report.

1. AbbVie
2. Adverum Biotechnologies
3. Anagenesis Biotechnologies
4. Asklepios BioPharmaceutical (acquired by Bayer)
5. Astellas Pharma
6. Batu Biologics
7. Bayer
8. Beam Therapeutics
9. Beth Israel Deaconess Medical Center 
10. Biogen
11. Biomedical Advanced Research and Development Authority (BARDA) 
12. BlueRock Therapeutics (acquired by Bayer)
13. Bristol-Myers Squibb
14. California Institute for Regenerative Medicine (CIRM)
15. CARB-X
16. Caribou Biosciences
17. Caspr Biotech
18. Catalent
19. Celgene
20. Cellectis
21. Chengdu MedGenCell
22. Columbia University
23. CRISPR Therapeutics
24. CureVac
25. Cystic Fibrosis Foundation Therapeutics
26. Duke University
27. DuPont
28. EdiGene
29. Editas Medicine
30. Eisai
31. Eligo Bioscience
32. EmendoBio (acquired by AnGes)
33. Excision BioTherapeutics
34. Eye & ENT Hospital of Fudan University
35. Fate Therapeutics
36. GEMoaB
37. GenEdit
38. Genevant Sciences
39. GlaxoSmithKline 
40. Graphite Bio
41. Guangzhou Anjie Biomedical Technology
42. Harvard University
43. ImmunoChina Pharmaceuticals
44. Institute of Molecular and Clinical Ophthalmology Basel (IOB)
45. Intellia Therapeutics
46. Intima Bioscience
47. J. David Gladstone Institutes
48. Janssen Pharmaceuticals
49. KSQ Therapeutics
50. Locus Biosciences
51. Mammoth Biosciences
52. Masonic Cancer Center
53. MaSTherCell
54. MaxCyte
55. Memorial Sloan Kettering Cancer Center
56. Modalis Therapeutics (formerly known as EdiGENE)
57. Mustang Bio
58. Nanjing Bioheng Biotech
59. National Institutes of Health
60. Navega Therapeutics
61. Nemesis Bioscience
62. Neon Therapeutics
63. Novartis
64. Poseida Therapeutics
65. Prime Medicine
66. ProBioGen
67. Refuge Biotechnologies
68. Regeneron
69. Sandhill Therapeutics
70. Sarepta Therapeutics
71. Scribe Therapeutics
72. Seven Therapeutics
73. Shanghai BD Gene Therapeutics
74. Shanghai Bioray Laboratories
75. Sherlock Biosciences
76. Shionogi
77. Sichuan University
78. SNIPR Biome
79. Spotlight Therapeutics
80. StrideBio
81. Takeda
82. Temple University
83. TeneoBio
84. The First Affiliated Hospital of Guangdong Pharmaceutical University
85. University of California
86. University of Texas
87. University of Washington
88. Vertex Pharmaceuticals
89. Verve Therapeutics
90. ViaCyte
91. Xijing Hospital
92. Yufan Biotechnologies
93. Zhejiang University