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CRISPR Based Therapeutics Market by Type of Therapy, Therapeutic Approach, Therapeutic Area, and Key Geographical Regions: Industry Trends and Global Forecasts 2021-2030

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  • 217 Pages
  • June 2021
  • Region: Global
  • Roots Analysis
  • ID: 5411389


Clustered regularly interspaced short palindromic repeats (CRISPR) are a family of DNA sequences, which constitute a primitive immune system that is responsible for protecting prokaryotic cells from phage infections. It was first described in 1987, however, its potential as a gene editing tool was not realized until 2012. Since then, the CRISPR revolution has not shown any signs of slowing down and has been responsible for significant advances in molecular biology and therapy development. Fundamentally, the CRISPR/Cas system involves specific palindromic DNA sequences which work in tandem with a family of caspase enzymes (Cas9, Cas12), in order to excise gene fragments with high precision. Compared to the other targeted nuclease-based systems, CRISPR is relatively faster, and cost-efficient; as a result, the demand for this gene editing tool is very high. The relatively recent discovery/development of novel accompanying nucleases, namely Cas12a, Cas13, Cas14 and dCas9, has significantly improved the precision of this technology. Presently, there are several companies using different variants of the CRISPR/Cas technology for basic research, and the development of gene editing solutions. However, the therapeutic use of this versatile genetic manipulation tool is only being investigated by a select few stakeholders in the pharmaceutical industry. The aforementioned scenario is attributed to the surrogate licensing model, which has granted exclusive control of the associated intellectual property (IP) to three leading players, namely Editas Medicine, CRISPR Therapeutics and Intellia Therapeutics, in the contemporary market.

Clinical trials of CRISPR based therapeutics are currently focused mainly on oncological and hematological disorders; however, several product candidates against certain neurological disorders and infectious diseases, specifically targeting recurrent conditions, are under investigation. Post 2014, the overall interest in this technology has grown exponentially, with several start-ups entering the market and 6 of the top 10 pharmaceutical companies restructuring their efforts in this direction. Over time, a substantial body of evidence has also been generated validating the therapeutic applications of this technology, which has, in turn, prompted the establishment of numerous strategic partnerships (focused on therapy development and clinical research) and has caused investors to put in significant capital into innovator companies involved in this domain, over the last two years alone. In fact, the three leading companies in this industry segment together have combined market capitalization of more than USD 10 billion, and have raised more than USD 2.8 billion in various funding rounds. Despite the possibly limitless potential of the CRISPR/Cas technology, further investigation, probing its safety and therapeutic efficacy in large diverse populations, is required. Key impediments to approval and other existing challenges that are being addressed by stakeholders, include off-target toxicity-related concerns and complexities related to the delivery of CRISPR components into target cells. Concerning delivery, innovators in this field have reported notable success using different types of platforms for facilitating the intracellular administration of CRISPR components; examples of successful delivery methods include electroporation, AAV vectors and lipid nanoparticles (LNPs). A few companies are also evaluating bacteriophages as a potential delivery system for such products. Promising clinical results, and ongoing technical developments, coupled to the growing interest of biopharmaceutical developers, are anticipated to push pipeline products to higher phases and on to commercialization. We believe that the market is likely to evolve at a commendable pace over the next decade.

Scope of the Report

The “CRISPR Based Therapeutics Market, 2021-2030” report features an extensive study of the current market landscape and future opportunity for the players involved in the development of CRISPR based therapeutics for the treatment of a variety of disease conditions. The study presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain, across different geographies. 

Amongst other elements, the report includes:

  • A review of the CRISPR 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. It also includes information on the completed, ongoing and planned clinical trials for CRISPR based therapeutics, sponsored by various industry players.
  • Elaborate profiles of key players in this domain. Each company profile features a brief overview of the company, its financial information (if available), a brief description of its therapeutic candidates, recent developments, and an informed future outlook.
  • An in-depth analysis of around 2,000 patents related to CRISPR technology that have been filed/granted, since 2015, highlighting the key trends associated with these patents, across type of patent, publication year and application year, regional applicability, IPCR symbols, emerging focus areas, inventor information, leading patent assignees (in terms of number of patents filed/granted), patent benchmarking and valuation. 
  • An analysis of the partnerships that have been inked by various stakeholders engaged in the development of CRISPR based therapeutics, during the period 2014-2020, 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.
  • An analysis of the investments made at various stages of development of the companies engaged in this field, covering instances of seed financing, venture capital financing, grants/awards, capital raised from IPOs and subsequent offerings.
  • An analysis of the start-ups (established in the time period between 2013-2020 and have less than 200 employees) engaged in the development of CRISPR based therapeutics, based on several parameters, such as number of candidates in discovery, preclinical and clinical phase of development, therapeutic area, amount raised through funding, number of investors, type of funding, number of deals signed, and number of patents filed.

One of the key objectives of the report was to estimate the future growth potential of CRISPR based therapeutics market, over the coming decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the financial evolution of the market for the period 2021-2030. For this purpose, we have segmented the future 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), [D] key geographical regions (North America, Europe and Asia-Pacific). To account for uncertainties and to add robustness to our model, we have provided three market forecast scenarios, portraying the conservative, base and optimistic tracks of the anticipated industry’s growth.

Key Questions Answered

  • Who are the leading players engaged in the development of CRISPR based therapeutics?
  • Which key clinical conditions can be treated by CRISPR based drugs?
  • What are the investment trends in this industry?
  • Which partnership models are commonly adopted by stakeholders engaged in this domain?
  • How has the intellectual property landscape in this market evolved over the years?
  • Which factors are likely to influence the evolution of this market?
  • How is the current and future market opportunity likely to be distributed across key market segments?

Table of Contents

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

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
3.3. CRISPR/Cas Component Delivery Vehicles
3.3.1. Physical Delivery
3.3.2. Viral Delivery
3.3.3. Non-Viral Delivery
3.4. Applications of the CRISPR/Cas System
3.5. CRISPR/Cas based Therapeutics
3.6. Challenges and Future Prospects

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

5.1. 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

6.1. 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

7.1. 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

8.1. 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

9.1. 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: Perspective

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) Target Patient Population Sales Forecast (USD Million)
10.9.2. OTQ923 / HIX763 (Intellia Therapeutics / Novartis) Target Patient Population Sales Forecast (USD Million)
10.9.3. CTX001 for Sickle Cell (CRISPR Therapeutics) Target Patient Population Sales Forecast (USD Million)
10.9.4. CTX001 for Transfusion-Dependent Beta Thalassemia (CRISPR Therapeutics) Target Patient Population Sales Forecast (USD Million)
10.9.5. Unnamed Drug for Metastatic Gastrointestinal Epithelial Cancer (Intima Bioscience) Target Patient Population Sales Forecast (USD Million)
10.9.6. BD111 (Shanghai BD Gene Therapeutics) Target Patient Population Sales Forecast (USD Million)
10.9.7. Unnamed Drug for Beta Thalassemia Major (Shanghai Bioray Laboratories) Target Patient Population Sales Forecast (USD Million)
10.9.8. CD19-UCART (Shanghai Bioray Laboratories) Target Patient Population Sales Forecast (USD Million)
10.9.9. Unnamed Drug for Advanced Non-Small Cell Lung Cancer (Guangzhou Anjie Biomedical Technology) Target Patient Population Sales Forecast (USD Million)
10.9.10. Unnamed Drug for Advanced Prostate Cancer (Guangzhou Anjie Biomedical Technology) Target Patient Population Sales Forecast (USD Million)

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


Companies Mentioned

  • AbbVie
  • Adverum Biotechnologies
  • Anagenesis Biotechnologies
  • Asklepios BioPharmaceutical (acquired by Bayer)
  • Astellas Pharma
  • Batu Biologics
  • Bayer
  • Beam Therapeutics
  • Beth Israel Deaconess Medical Center 
  • Biogen
  • Biomedical Advanced Research and Development Authority (BARDA) 
  • BlueRock Therapeutics (acquired by Bayer)
  • Bristol-Myers Squibb
  • California Institute for Regenerative Medicine (CIRM)
  • CARB-X
  • Caribou Biosciences
  • Caspr Biotech
  • Catalent
  • Celgene
  • Cellectis
  • Chengdu MedGenCell
  • Columbia University
  • CRISPR Therapeutics
  • CureVac
  • Cystic Fibrosis Foundation Therapeutics
  • Duke University
  • DuPont
  • EdiGene
  • Editas Medicine
  • Eisai
  • Eligo Bioscience
  • EmendoBio (acquired by AnGes)
  • Excision BioTherapeutics
  • Eye & ENT Hospital of Fudan University
  • Fate Therapeutics
  • GEMoaB
  • GenEdit
  • Genevant Sciences
  • GlaxoSmithKline 
  • Graphite Bio
  • Guangzhou Anjie Biomedical Technology
  • Harvard University
  • ImmunoChina Pharmaceuticals
  • Institute of Molecular and Clinical Ophthalmology Basel (IOB)
  • Intellia Therapeutics
  • Intima Bioscience
  • J. David Gladstone Institutes
  • Janssen Pharmaceuticals
  • KSQ Therapeutics
  • Locus Biosciences
  • Mammoth Biosciences
  • Masonic Cancer Center
  • MaSTherCell
  • MaxCyte
  • Memorial Sloan Kettering Cancer Center
  • Modalis Therapeutics (formerly known as EdiGENE)
  • Mustang Bio
  • Nanjing Bioheng Biotech
  • National Institutes of Health
  • Navega Therapeutics
  • Nemesis Bioscience
  • Neon Therapeutics
  • Novartis
  • Poseida Therapeutics
  • Prime Medicine
  • ProBioGen
  • Refuge Biotechnologies
  • Regeneron
  • Sandhill Therapeutics
  • Sarepta Therapeutics
  • Scribe Therapeutics
  • Seven Therapeutics
  • Shanghai BD Gene Therapeutics
  • Shanghai Bioray Laboratories
  • Sherlock Biosciences
  • Shionogi
  • Sichuan University
  • SNIPR Biome
  • Spotlight Therapeutics
  • StrideBio
  • Takeda
  • Temple University
  • TeneoBio
  • The First Affiliated Hospital of Guangdong Pharmaceutical University
  • University of California
  • University of Texas
  • University of Washington
  • Vertex Pharmaceuticals
  • Verve Therapeutics
  • ViaCyte
  • Xijing Hospital
  • Yufan Biotechnologies
  • Zhejiang University