Top Gene Editing Companies
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Published: April 2024
Driven by numerous developmental breakthroughs demonstrating the vast potential of cell and gene therapy in the treatment of various genetic disorders, gene editing tools have garnered considerable attention in the genome editing market. We analyzed over 110 gene editing companies that are engaged in offering genome editing technologies. In this article, discover 8 top gene editing companies and their capabilities in this industry.
Driven by numerous developmental breakthroughs and the results of studies demonstrating the vast potential of gene therapy and cell therapies made possible through genome editing technologies, this therapeutic modality has been portrayed as next generation of medicine for the treatment of various diseases. Such transformational therapies have the capability to offer enormous clinical benefits and significantly improve the overall quality of life. As of 2023, globally, over 1,000 cell and gene therapy candidates are currently being investigated by various stakeholders in the pharmaceutical domain. The ongoing research activities focused on these therapies have further fueled the demand for gene editing technologies that can result in to-the-point modifications of genome sequences.
The gene editing tools have been extensively used as corrective strategies to treat the clinical conditions which develop as a result of genetic abnormalities, such as sickle cell disease, Parkinson’s disease, hearing loss, peripheral artery disease, spinal muscular atrophy, autoimmune diseases, and other genetic disorders. It is worth highlighting that prophylaxis treatment is another segment of the gene editing applications that has garnered significant attention in the gene editing market. However, the development of gene / genome edited therapy involves a heavy investment for drug discovery, development, and manufacturing in the gene research industry. Of all the genome editing tools, CRISPR technology, specifically, CRISPR-Cas9 has emerged as the leading genetic engineering technique developed by the stakeholders in the gene editing market. Recently, the domain has witnessed a paradigm shift from conventional CRISPR technology to base editing and prime editing techniques. These novel tools are aimed to improve the long-term safety and efficiency of gene delivery. Given the ongoing pace of innovation in this field, including encouraging results in clinical trials and accelerated drug approvals, the genome editing market is likely to witness significant market growth during the forecast period.
Roots Analysis has conducted an exhaustive study on Genome Editing Market featuring the current market landscape and future opportunity for the gene editing companies that are engaged in the development of genome editing technologies, over a span of 12 years.
Table of Contents
Below, we have listed (in alphabetical order), the top 8 gene editing companies.
- Arcturus Therapeutics
- Beam Therapeutics
- Caribou Biosciences
- Century Therapeutics
- CRISPR Therapeutics
- Editas Medicine
- Intellia Therapeutics
- Prime Medicine
Interested in exploring all 110+ gene editing companies and their recent initiatives?
Discover Below the Details on 8 Top Gene Editing Companies
Let us deep dive to understand the activity of the 8 top gene editing companies (shortlisted on the basis of funding raised in capital investment (more than USD 490 million)) in the genome editing market. It is essential to note that the selection of top players can differ substantially according to the defined criteria.
With Vast Experience in RNA-based Therapeutics, Arcturus Therapeutics is Engaged in Development of Therapeutics for Rare Diseases and Vaccines
1. Arcturus Therapeutics
Arcturus Therapeutics is a US-based company that specializes in the development of mRNA therapeutics through its lipid nanoparticles (LNP) based delivery platforms, LUNAR® and STARR™. The company claims to leverage these technologies for gene upregulation, gene editing and gene silencing. In addition, the company is also engaged in the development of mRNA vaccines for the treatment of infectious diseases (coronavirus, seasonal and pandemic influenza).
Arcturus Therapeutics: Company Details
| Key Parameters | Description |
| Company Logo |  |
| Year of Establisment | 2013 |
| Number of Employees | 51-200 |
| Headquarter |  |
| Funding Amount (as of July 2023) | USD 622 Million |
Arcturus Therapeutics: Gene Editing Technology Portfolio
| Key Parameters | LUNAR |
| Type of Gene Editing Technique | CRISPR-Cas system, TALENs, Meganucleases |
| Gene Editing Approach | Knock-in, Knock-out |
| Type of Gene Delivery Method | In-vivo |
| Type of Gene Delivery Modality | LNPs |
| Route of Administration | Intravenous |
| Therapeutic Areas | Infectious Diseases |
| Highest Phase of Drug Development Supported | Clinical |
Partnership Signed by Arcturus Therapeutics Focusing on Gene Editing Domain: In November 2022, the company entered into a collaboration with CSL Seqirus in order to license its LUNAR platform for the development of vaccines for infectious diseases.
Further details on recent initiatives taken by Arcturus Therapeutics as well as other gene editing companies have been provided in detail in the report.
Beam Therapeutics is Focused on the Development of Best-in-Class Genetic Medicines based on Base Editing Approach
2. Beam Therapeutics
Beam Therapeutics is a biotechnology company involved in the development of precision therapeutic products for serious disease indications using its proprietary next-generation CRISPR technologies, which are capable of making precise edits to single base pairs in DNA and RNA. These technologies are being designed to offer various types of modifications, including gene activation, gene knock-out, gene correction, surface protein modification and multiplex gene editing. It is worth highlighting that the company owns a 100,000 square feet cGMP certified clinical / commercial facility in North Carolina.
Beam Therapeutics: Company Details
| Key Parameters | Description |
| Company Logo |  |
| Year of Establisment | 2017 |
| Number of Employees | 501-1,000 |
| Headquarter |  |
| Funding Amount (as of July 2023) | USD 790 Million |
Beam Therapeutics: Gene Editing Technology Portfolio
| Key Parameters | Beam's Base Editing Technology | Beam's CRISPR Cas12b Nuclease Technoloy |
| Type of Gene Editing Technique | CRISPR-Cas system (Base editing) | CRISPR-Cas system (Base editing) |
| Gene Editing Approach | Knock-in | Knock-in, Knock-out |
| Type of Gene Delivery Method | In-vivo, Ex-vivo | Ex-vivo |
| Type of Gene Delivery Modality | Adeno-associated viral (AAV) vectors, LNPs, mRNA, T-cells, Stem cells |
T-cells |
| Route of Administration | Intravenous | Intravenous |
| Therapeutic Areas | Hematological Disorders, Ophthalmological Disorders, Metabolic Disorders, Genetic Disorders, Complement-driven Diseases |
Oncological Disorders, Neurological Disorders, Metabolic Disorders, Inflammatory Diseases |
| Highest Phase of Drug Development Supported |
Clinical | NA |
Partnership Signed by Beam Therapeutics Focusing on Gene Editing Domain: In September 2022, the company entered into a collaboration with Orbital Therapeutics, in order to license its non-viral delivery technology for research and development of various gene-editing based therapies.
Further details on recent initiatives taken by Beam Therapeutics as well as other gene editing companies have been provided in detail in the report.
Leveraging Various Genome Editing Strategies, Caribou Biosciences is Currently Conducting Clinical Trials for its Off-the-Shelf Cell Therapies
3. Caribou Biosciences
Caribou Biosciences is a clinical-stage biopharmaceutical company primarily engaged in the development of allogenic CAR-T and CAR-NK cell therapies. The company is also advancing its product pipeline into clinical phases of development for various hematological malignancies. In addition, the company claims to have a robust intellectual portfolio of CRISPR technology, that has been licensed from The Regents of the University of California and University of Vienna.
Caribou Biosciences: Company Details
| Key Parameters | Description |
| Company Logo |  |
| Year of Establisment | 2012 |
| Number of Employees | 51-200 |
| Headquarter |  |
| Funding Amount (as of July 2023) | USD 589 Million |
Caribou Biosciences: Gene Editing Technology Portfolio
| Key Parameters | Cas12a chRDNA Technology |
| Type of Gene Editing Technique | CRISPR-Cas system |
| Gene Editing Approach | Knock-in, Knock-out |
| Type of Gene Delivery Method | In-vivo, Ex-vivo |
| Type of Gene Delivery Modality | AAV vectors, CAR-T cells, CAR-NK cells, Other T-cells |
| Route of Administration | Intravenous |
| Therapeutic Areas | Oncological Disorders, Hematological Disorders |
| Highest Phase of Drug Development Supported | Clinical |
Investment secured by Caribou Biosciences to Aid Current / Future Operations: In 3Q 2023, the company secured capital investment worth USD 134.6 million through secondary offering.
Further details on recent initiatives taken by Caribou Biosciences as well as other gene editing companies have been provided in detail in the report.
Century Therapeutics is Committed to the Engineering of the First Allogeneic Cell Therapy Candidate With Six Precision Gene Edits
4. Century Therapeutics
Century Therapeutics is a biotechnology company engaged in the allogeneic cell therapy domain. The company’s extensive portfolio comprises of protein engineering and gene editing for the generation of induced pluripotent stem cells (iPSC)-derived cell therapies for hematological and solid tumor malignancies. It is worth mentioning that the company leverages its proprietary Allo-Evasion™ technology in order to design the cells that can evade immune host rejection.
Century Therapeutics: Company Overview
| Key Parameters | Description |
| Company Logo |  |
| Year of Establisment | 2018 |
| Number of Employees | 201-500 |
| Headquarter |  |
| Funding Amount (as of July 2023) | USD 691 Million |
Century Therapeutics: Gene Editing Technology Portfolio
| Key Parameters | Efficient Precision Gene Editing Technology |
| Type of Gene Editing Technique | CRISPR-Cas system |
| Gene Editing Approach | Knock-in, Knock-out |
| Type of Gene Delivery Method | In-vivo, Ex-vivo |
| Type of Gene Delivery Modality | Stem cells, NK cells, T-cells |
| Route of Administration | Intravenous |
| Therapeutic Areas | Oncological Disorders |
| Highest Phase of Drug Development Supported | Clinical |
Partnership Signed by Century Therapeutics on Gene Editing Domain: In January 2022, The company entered into a collaboration with Bristol-Myers Squibb in order to license its efficient precision gene editing technology for the development of cell therapies for cancer.
Further details on recent initiatives taken by Century Therapeutics as well as other gene editing companies have been provided in detail in the report.
CRISPR Therapeutics, a Venture Backed Company having Expertise in Gene Edited Therapies
5. CRISPR Therapeutics
CRISPR Therapeutics is a biopharmaceutical company focused on the development of transformative gene-based medicines for serious diseases, particularly somatic (non-germline) diseases. The company has its headquarters in Basel, Switzerland and operates through several centers located across the globe, such as London and Massachusetts. The company claims to be actively working on four focus areas; these include gene-edited hematopoietic stem cells, immune-oncology, gene editing, and regenerative medicine. It is worth mentioning that the firm states to leverage its proprietary CRISPR / Cas9 gene editing platform for the development of gene therapy products.
CRISPR Therapeutics: Company Overview
| Key Parameters | Description |
| Company Logo |  |
| Year of Establisment | 2013 |
| Number of Employees | 201-500 |
| Headquarter |  |
| Funding Amount (as of July 2023) | USD 1,097 Million |
CRSIPR Therapeutics: Gene Editing Technology Portfolio
| Key Parameters | CRISPR Technology Platform |
| Type of Gene Editing Technique | CRISPR-Cas system |
| Gene Editing Approach | Knock-in, Knock-out |
| Type of Gene Delivery Method | In-vivo, Ex-vivo |
| Type of Gene Delivery Modality | AAV vectors, LNPs, mRNA, Stem cells, CAR-T cells, CAR-NK cells |
| Route of Administration | Intravenous |
| Therapeutic Areas | Oncological Disorders, Hematological Disorders, Immunological Disorders, Ophthalmological Disorders, Neurological Disorders, Metabolic Disorders,Genetic Disorders, Muscle-related Disorders, Cardiovascular Disorders, Pulmonary Disorders |
| Highest Phase of Drug Development Supported | Clinical |
Partnership Signed by CRISPR Therapeutics Focusing on Gene Editing Domain: In March 2023, the company entered into a collaboration with Vertex Therapeutics, in order to license its CRISPR technology platform for research and development of cell therapy for type 1 diabetes.
Further details on recent initiatives taken by CRISPR Therapeutics as well as other gene editing companies have been provided in detail in the report.
Strengthened by a Robust Intellectual Property Portfolio, Editas Medicine Envisions To Pioneer in the Field of Genomic Medicine
6. Editas Medicine
Editas Medicine is a biotechnology company which claims to have expertise in the field of genome editing. The company utilizes its Cas9 and Cas12a technologies for the development of next-generation genomic medicines (in-vivo and ex-vivo) for oncological and hematological disorders. In addition, the company is also working in the field of plant gene editing, regenerative medicine and drug discovery. It is worth highlighting that the company’s operational models are based on technology licensing and collaborative product development agreements with other stakeholders in the industry.
Editas Medicine: Company Overview
| Key Parameters | Description |
| Company Logo |  |
| Year of Establisment | 2013 |
| Number of Employees | 201-500 |
| Headquarter |  |
| Funding Amount (as of July 2023) | USD 538 Million |
Editas Medicine: Gene Editing Technology Portfolio
| Key Parameters | SLEEK Gene Editing |
| Type of Gene Editing Technique | CRISPR-Cas system |
| Gene Editing Approach | Knock-in |
| Type of Gene Delivery Method | In-vivo, Ex-vivo |
| Type of Gene Delivery Modality | Stem cells, T cells, NK cells |
| Route of Administration | Intravenous |
| Therapeutic Areas | Oncological Disorders, Hematological Disorders, Immunological Disorders, Ophthalmological Disorders, Genetic Disorders |
| Highest Phase of Drug Development Supported | Clinical |
Partnership Signed by Editas Medicine Focusing on Gene Editing Domain: In January 2023, The company entered into a collaboration with Shoreline Biosciences in order to license its SLEEK Gene Editing platform for the development of Shoreline’s iNK Platform for tumors.
Further details on recent initiatives taken by Editas Medicine as well as other gene editing companies have been provided in detail in the report.
CRISPR-based Differentiated Technologies are the Cornerstones at Intellia Therapeutics for the Investigation of Patient-Focused Treatment
7. Intellia Therapeutics
Intellia Therapeutics is engaged in the development of in-vivo and ex-vivo CRISPR-based therapeutics for the treatment of various chronic diseases, including genetic, oncological, and immunological disorders. The firm is currently accelerating its candidates on to the clinical stage of development. In fact, the Intellia Therapeutics claims to be the first company to demonstrate the successful systemic CRISPR delivery in a human body. It is worth mentioning that the company’s co-founder Jennifer Doudna was awarded a Nobel Prize in Chemistry in 2020. The prize was presented to her for the development of CRISPR / Cas9 genome editing technology. This revolutionary work paved way for the precise, effective treatment of various intractable diseases.
Intellia Therapeutics: Company Details
| Key Parameters | Description |
| Company Logo |  |
| Year of Establisment | 2014 |
| Number of Employees | 201-500 |
| Headquarter |  |
| Funding Amount (as of July 2023) | USD 1,316 Million |
Intellia Therapeutics: Gene Editing Technology Portfolio
| Key Parameters | In-vivo CRISPR / Cas9 Genome Editing Platform | In-vivo CRISPR / Cas9 Genome Editing Platform |
| Type of Gene Editing Technique | CRISPR-Cas system (Base editing) | CRISPR-Cas system (Base editing) |
| Gene Editing Approach | Knock-in | Knock-in, Knock-out |
| Type of Gene Delivery Method | In-vivo, Ex-vivo | Ex-vivo |
| Type of Gene Delivery Modality | AAV vectors, LNPs | CAR-T cells, Stem cells |
| Route of Administration | Intravenous | NA |
| Therapeutic Areas | Hematological Disorders, Ophthalmological Disorders, Genetic Disorders, Lung-related Disorders, Liver-related Disorders |
Oncological Disorders, Immunological Disorders, Autoimmune Disorders, Inflammatory Diseases |
| Highest Phase of Drug Development Supported |
Clinical | Preclinical |
Partnership Signed by Intellia Therapeutics Focusing on Gene Editing Domain: In February 2022, the company entered into a collaboration with ONK Therapeutics in order to license its ex-vivo CRISPR/Cas9-based genome editing platform and LNP-based delivery technologies for the development of allogeneic NK cell therapies.
Further details on recent initiatives taken by Intellia Therapeutics as well as other gene editing companies have been provided in detail in the report.
Known as a Next-generation Gene Editing Technology Developer, Prime Medicine Strives to Shape the Future of Gene Editing Industry
8. Prime Medicine
Prime Medicine is a biotechnology company that specializes in the field of prime editing. Currently, the company’ pipeline consists of 18 programs being developed for the treatment of various indications, including cystic fibrosis, Duchenne muscle dystrophy, Friedreich’s ataxia, fragile X syndrome and Huntington’s disease. Fragile X Syndrome. It is worth highlighting that the company owns three manufacturing facilities in Cambridge and one chemistry facility in Watertown.
Prime Medicine: Company Details
| Key Parameters | Description |
| Company Logo |  |
| Year of Establisment | 2019 |
| Number of Employees | 201-500 |
| Headquarter |  |
| Funding Amount (as of July 2023) | USD 495 Million |
Prime Medicine: Gene Editing Technology Portfolio
| Key Parameters | Prime Editing Technology | PASSIGE Platform |
| Type of Gene Editing Technique | CRISPR-Cas system (Prime editing) | CRISPR-Cas system (Prime editing) |
| Gene Editing Approach | Knock-in, Knock-out | Knock-in, Knock-out |
| Type of Gene Delivery Method | In-vivo, Ex-vivo | In-vivo |
| Type of Gene Delivery Modality | AAV vectors, LNPs, T-cells, Stem cells | T-cells |
| Route of Administration | Intravenous | Intravenous |
| Therapeutic Areas | Hematological Disorders, Ophthalmological Disorders, Neurological Disorders, Metabolic Disorders, Genetic Disorders, Muscle-related Disorders, Hepatic Disorders, Otic Disorders |
Oncological Disorders, Immunological Disorders, Metabolic Disorders, Genetic Disorders |
| Highest Phase of Drug Development Supported |
Preclinical | Preclinical |
Partnership Signed by Prime Medicine Focusing on Gene Editing Domain: In June 2023, the company entered into a research and development agreement with Cimeio Therapeutics in order to advance its prime editing platform.
Further details on recent initiatives taken by Prime Medicine as well as other gene editing companies have been provided in detail in the report.
What About the Other Gene Editing Companies Around the World?
The above presentation features eight top gene editing companies selected from a pool of over 110 players that we have compiled. If you're interested, you can download the Sample Report on this topic by Roots Analysis. For personalized assistance in identifying the most relevant solutions based on your specific criteria, please don't hesitate to reach out to us at sales@rootsanalysis.com.
Sources
- https://ir.arcturusrx.com/news-releases/news-release-details/arcturus-announces-collaboration-csl-develop-and-commercialize
- https://in.marketscreener.com/quote/stock/BEAM-THERAPEUTICS-INC-102685993/news/Beam-Therapeutics-Inc-Enters-into-License-and-Research-Collaboration-Agreement-with-Orbital-Therape-41722395/
- https://investor.cariboubio.com/news-releases/news-release-details/caribou-biosciences-reports-second-quarter-2023-financial
- https://news.bms.com/news/details/2022/Century-Therapeutics-and-Bristol-Myers-Squibb-Enter-into-a-Strategic-Collaboration-to-Develop-iPSC-derived-Allogeneic-Cell-Therapies/default.aspx
- https://investors.vrtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-announce-licensing-agreement
- https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-and-shoreline-biosciences-enter-definitive
- https://ir.intelliatx.com/news-releases/news-release-details/intellia-and-onk-therapeutics-announce-collaboration-advance
- https://investors.primemedicine.com/news-releases/news-release-details/prime-medicine-and-cimeio-therapeutics-announce-research