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Global CRISPR Cas9 Market, Clinical Trials & Therapeutic Application Outlook 2024

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  • October 2019
  • Region: Global
  • Kuick Research
  • ID: 4846783

Report Highlights

  • Report Focuses On Human Clinical Trials & Human Therapeutic Application
  • Global CRISPR Cas9 Market Overview
  • Global CRISPR Cas9 Clinical Trials: More Than 40 Trials
  • Global CRISPR Cas9 Clinical Trials by Company & Indication
  • Global CRISPR Cas9 Market Regional Scenario
  • Global CRISPR Cas9 Therapy Application by Multiple Indications
  • Key Companies Profile Involved In Clinical Trials: 12 Companies

CRISPR technology is one of the most advanced technologies falling under the category of gene editing. It stands out as a mature existing system that can be used to engineer the genes with the desired ones. CRISPR contains innovative capabilities making its potential benefits endless. Till now, the theoretical knowledge about CRISPR has gained much attention and now it’s the time for us to apply it for practical use.

CRISPR is one such technology that has totally revolutionized the working process in the scientific field. It has been only a few years since its discovery and CRISPR has started dominating the research labs all over the world. In spite of being a newly discovered technology, CRISPR has come out with the latest methodologies of treating diseases with genetic or lifestyle inherited diseases.

Making targeted changes to the genome of an individual which was a long-standing goal for the researchers, has now become a dream come like a true situation. The development of CRISPR technology has helped the bio-medical researchers to put out any experiment in an efficient way. CRISPR is not the first tool to get discovered for performing gene editing but it has made its way all to the top of the list in gene-editing technologies.

CRISPR technology comes with a lot of potential in it. It is the most advanced technology for treating deadly diseases that mankind is suffering from. CRISPR is successful in showing positive results in curing diseases such as sickle cell anemia, cancer, hepatitis B and cholesterol. CRISPR is used for producing alterations in the somatic cell lines as well as the germline (reproductive cells). With this application of CRISPR, it is possible to correct the mutation residing inside the genome of a fetus.

Clinical trials about the CRISPR components help in achieving the efficacy of the technology. More than 30 CRISPR-Cas9 clinical trials are going on, with a participation of more than 1,000 patients. Among all the countries, the US and China are the most active countries that are running the maximum number of trials. CRISPR has not only marked its presence in the pharma sector, but it has also come across a long path in gaining respect in the agriculture field. There is constant innovation going on in the laboratories for attaining maximum benefits from the CRISPR components.

The increasing potential benefits received from CRISPR are creating an unimaginable effect on the market of CRISPR across the globe. The market of CRISPR is going through giant progress, as the benefits from CRISPR are increasing day by day. The market of CRISPR has shown a tremendous jump over the last few years. Researchers are also exploring different ways of utilizing CRISPR in both diagnostic as well as therapeutic development.

The successful commercialization of such development is likely to drive the market’s revenue growth. The market of CRISPR is also driven by the increasing number of cancer cases, autoimmune diseases in the world. In spite of the increasing cancer cases around the globe, other major factors driving the growth of CRISPR are the leading manufacturers of the pharmaceutical industry, technological advancements and the presence of skilled researchers.

Table of Contents

1. Introduction to CRISPR-Cas9
1.1 Overview
1.2 History of CRISPR-Cas9 Technology

2. Mechanism of Action
2.1 Immune Defense with CRISPR in Single-Celled Microorganisms
2.2 CRISPR Technology

3. Delivery Methods for CRISPR-Cas9
3.1 Delivery of CRISPR-Cas9 components through Physical Methods
3.1.1 Microinjection
3.1.2 Electroporation
3.1.3 Hydrodynamic Delivery
3.2 Delivery of CRISPR-Cas9 Components through Viral Vectors
3.2.1 Adeno-Associated Virus (AAV)
3.2.2 Lentivirus & Adenovirus
3.3 Delivery of CRISPR-Cas9 Components through Non-Viral Vectors (Biomaterials)
3.3.1 Liposomes
3.3.2 Gold Nanoparticles
3.3.3 DNA Nanoclew

4. CRISPR-Cas9: A Home to a Variety of Applications
4.1 Advantages Accompanying CRISPR-Cas9 Technology
4.2 CRISPR-Cas9 Comparison with other Genome Editing Tools

5. Global CRISPR-Cas9 Clinical Trials by Company & Indication
5.1 Research
5.2 Preclinical
5.3 Phase-I
5.4 Phase-I/II
5.5 Phase-II

6. Global CRISPR-Cas9 Market Current Overview
6.1 Global CRISPR Market Analysis
6.2 Global CRISPR Market Regional Scenario

7. Elimination of Genetic Disease:  Sickle-Cell Anemia through CRISPR-Cas9
7.1 Overview
7.2 Sickle Cell Treatment with CRISPR
7.3 Clinical Trial for Testing CRISPR against Sickle Cell Anemia

8. Revolutionizing the Treatment of Beta-Thalassemia
8.1 Overview
8.2 CRISPR Application in Treating Beta-Thalassemia
8.3 CRISPR Clinical Trial for Beta-Thalassemia

9. Potential for Curing Cancer
9.1 Overview
9.2 CRISPR-Cas9 Fight for Cancer
9.3 CRISPR Trial for Cancer Treatment Research

10. CRISPR-Cas9: A Step Closer to Treat Huntington’s Disease
10.1 Overview
10.2 CRISPR-Cas9 Towards Huntington’s Disease
10.3 CRISPR Mediated Treatment of Huntington’s Disease in Mouse Models

11. CRISPR-Cas9 Technique to Reverse Obesity Epidemic (Type 2 Diabetes)
11.1 Overview
11.2 CRISPR Treatment for Obesity
11.3 Clinical Trials for Reducing Obesity Using CRISPR

12. CRISPR-Cas9 Treatment for Cholesterol
12.1 Overview
12.2 Expanding the Application of CRISPR for Cholesterol
12.3 Lab  Clinical Studies Reflecting the Positive Side of CRISPR

13. CRISPR-Cas9 Against Malaria
13.1 Overview
13.2 CRISPR-Cas9 Technology for Malaria Treatment
13.3 Mosquitoes Carrying Parasites Eliminated using CRISPR-Cas9

14. CRISPR-Cas9 for Knocking Out Cystic Fibrosis
14.1 Overview
14.2 CRISPR-Cas9: A Versatile Tool for Deactivating CFTR
14.3 Development & Research: CRISPR-Cas9

15. CRISPR-Cas9 for the Removal of Hepatitis B
15.1 Overview
15.2 CRISPR-Cas9 Rising against cccDNA
15.3 Clinical Lab Studies Clears HBV cccDNA on Human Cell Lines

16. Permanent Cure for HIV/AIDS Treatment
16.1 Overview
16.2 CRISPR-Cas9 for Deleting CCR5 Gene
16.3 Clinical Results Achieved Using CRISPR-Cas9 for HIV/AIDS
16.4 Gene-Edited Babies: HIV resistant

17. CRISPR Hope for Alzheimer’s Disease
17.1 Overview
17.2 CRISPR-Cas9 for Alzheimer’s Disease
17.3 Lab Clinical Studies for Eliminating Alzheimer’s

18. CRISPR-Cas9 to Correct Duchenne Muscular Dystrophy (DMD)
18.1 Overview
18.2 CRISPR-Cas9 Technology Over-Powering DMD Gene
18.3 CRISPR Success in Treating DMD in Mice Models

19. CRISPR Enhances the Reach of Immunotherapy
19.1 CRISPR for Immunosuppressive Defects
19.2 CRISPR Mediated Generation of CAR-T Cells

20. Global CRISPR-Cas9 Market Dynamics
20.1 Market Drivers for CRISPR-Cas9 Therapy
20.2 Challenges Overpowering CRISPR

21. Global CRISPR-Cas9 Market  Future Outlook

22. Competitive Landscape
22.1 Beam Therapeutics
22.2 Casebia Therapeutics
22.3 CRISPR Therapeutics
22.4 EdiGENE Corporation
22.5 Editas Medicine
22.6 Emendo Biotherapeutics
22.7 Intellia Therapeutics
22.8 Modalis Therapeutics
22.9 Neon Therapeutics
22.10 Refuge Biotechnologies
22.11 Sarepta Therapeutics
22.12 Vertex Pharmaceuticals

List of Figures
Figure 1-1: Key Events in the History of CRISPR-Cas9 System Development
Figure 2-1: CRISPR Immune Mechanism in Bacteria
Figure 2-2: CRISPR Technology Working in Cell Lines
Figure 3-1: Delivery Methods for CRISPR-Cas9
Figure 3-2: Delivery Pathways for CRISPR-Cas9 Components
Figure 3-3: Physical Mode of Delivery for CRISPR-Cas9 Components
Figure 3-4: Viral Mode of Delivery for CRISPR-Cas9 Components
Figure 3-5: Non-Viral Mode of Delivery for CRISPR-Cas9 Components
Figure 4-1: Advantages of CRISPR-Cas9 Technology
Figure 4-2: Comparison between Different Genome Editing Tools
Figure 5-1: Global - CRISPR-Cas9 Clinical Trials by Phase (%), 2019 till 2024
Figure 5-2: Global - CRISPR-Cas9 Clinical Trials by Phase (Number), 2019 till 2024
Figure 6-1: Global - CRISPR Investments (US$ Million), 2011 & 2016
Figure 6-2: Global - Research Papers Published, 2011 & 2016
Figure 6-3: Global - CRISPR Market Size (US$ Million), 2018 - 2024
Figure 7-1: Normal RBCs VS Sickle Shaped RBCs
Figure 7-2: Treatment of Sickle Cell Anemia through CRISPR-Cas9
Figure 7-3: Phases Involved in a Clinical Trial of a Drug
Figure 8-1: Mutated Gene leading to Beta-Thalassemia
Figure 8-2: CRISPR-Cas9 Therapy for Beta-Thalassemia
Figure 9-1: Difference between Normal Cells & Cancer Cells
Figure 9-2: CRISPR-Cas9 Treatment for Cancer
Figure 9-3: Ongoing Clinical Trials on CRISPR in Different Types of Cancer
Figure 10-1: Defective Genes Leading to Huntington’s Disease
Figure 10-2: CRISPR-Cas9 Technique for Huntington’s Disease
Figure 10-3: Experiment Conducted to Test CRISPR-Cas9 Effect on Transgenic R6/2 Mouse Model
Figure 11-1: Obesity-Related Health Issues
Figure 11-2: Mutations Leading to Obesity
Figure 11-3: CRISPR-Cas9 Controlling Obesity
Figure 12-1: Cholesterol Pathways Present in the Body
Figure 12-2: Mutation Leading to Clogging of Arteries
Figure 12-3: CRISPR-Cas9 against Cholesterol
Figure 12-4: Experiment Conducted to Test CRISPR-Cas9 on Cholesterol Level
Figure 13-1: Malaria Transmission Cycle
Figure 13-2: CRISPR Cas9 Machinery against Malaria
Figure 13-3: Experiment Conducted to Test CRISPR against Plasmodium
Figure 14-1: CRISPR-Cas9 Correcting Cystic Fibrosis
Figure 14-2: Experiment Conducted on CF Patient to Evaluate CRISPR
Figure 15-1: HBV Virus Infecting Healthy Liver Cells
Figure 15-2: CRISPR Activity against Hepatitis B Virus
Figure 15-3:  Experiment Showing Reduction in HBV Viral Proteins due to CRISPR
Figure 16-1: HIV Infection Leading to AIDS
Figure 16-2: CRISPR Knocking Out CCR5 Gene
Figure 17-1: APP Gene Mutation Leading to Alzheimer’s Disease
Figure 17-2: CRISPR-Cas9 Machinery for Alzheimer’s Disease
Figure 17-3: Strategies Adopted by Researchers to Treat Alzheimer’s Through CRISPR
Figure 17-4: Experiment Conducted to Test CRISPR for Alzheimer’s
Figure 18-1: DMD Gene Mutation Leading to Duchenne muscular Dystrophy
Figure 18-2: CRISPR-Cas9 Editing DMD Gene
Figure 18-3: Experiment Showing the Success of CRISPR in Treating DMD in Mice
Figure 19-1: Cancer Cells Escaping Immune
Figure 19-2: CRISPR Potential Activities
Figure 20-1: Key Drivers Leading to the Development of CRISPR
Figure 21-1: Future of CRISPR-Cas9 Gene Editing Technology

Companies Mentioned

A selection of companies mentioned in this report includes:

  • Beam Therapeutics
  • Casebia Therapeutics
  • CRISPR Therapeutics
  • EdiGENE Corporation
  • Editas Medicine
  • Emendo Biotherapeutics
  • Intellia Therapeutics
  • Modalis Therapeutics
  • Neon Therapeutics
  • Refuge Biotechnologies
  • Sarepta Therapeutics
  • Vertex Pharmaceuticals