Top Gene Editing Companies

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.

  1. Arcturus Therapeutics
  2. Beam Therapeutics
  3. Caribou Biosciences
  4. Century Therapeutics
  5. CRISPR Therapeutics
  6. Editas Medicine
  7. Intellia Therapeutics
  8. Prime Medicine

Top 8 Gene Editing Companies

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 Arcturus Therapeutics, Gene Editing Companies
Year of Establisment   2013
Number of Employees  51-200
Headquarter Arcturus Therapeutics, 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 Beam Therapeutics, Gene Editing Companies
Year of Establisment   2017
Number of Employees  501-1,000
Headquarter Beam Therapeutics, 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 Caribou Biosciences, Gene Editing Companies
Year of Establisment   2012
Number of Employees  51-200
Headquarter Caribou Biosciences, 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 Century Therapeutics, Gene Editing Companies
Year of Establisment   2018
Number of Employees  201-500
Headquarter Century Therapeutics, 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 CRISPR Therapeutics, Gene Editing Companies
Year of Establisment   2013
Number of Employees  201-500
Headquarter CRISPR Therapeutics, 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 Editas Medicine, Gene Editing Companies
Year of Establisment   2013
Number of Employees  201-500
Headquarter Editas Medicine, 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 Intellia Therapeutics, Gene Editing Companies
Year of Establisment   2014
Number of Employees  201-500
Headquarter Intellia Therapeutics, 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 Prime Medicine, Gene Editing Companies
Year of Establisment   2019
Number of Employees  201-500
Headquarter Prime Medicien, 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.

About Author

Prachi_Bhalla

Prachi holds an M.Tech degree in Biotechnology from Indian Institute of Technology, Guwahati and B.E. degree in Biotechnology from Panjab University, Chandigarh. Upon completing her postgraduate studies, she embarked on her research career by securing the position of Senior Research Fellow at PGIMER, Chandigarh. In her more than two years of scientific career, she honed her skills in the field of cell and molecular biology, pathology and organic chemistry. Prachi has over two years of remarkable experience in the business research and consulting domain. She is equipped with the distinctive skill set to thrive and contribute immensely to the development of syndicate reports and assignments pertaining to the pharmaceutical and biotechnology industry. She has persistently sought to present impactful, critical insights to the clients through several drug-based, product-based, technology-based and service-based research reports.

Sources

  1. https://ir.arcturusrx.com/news-releases/news-release-details/arcturus-announces-collaboration-csl-develop-and-commercialize
  2. 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/
  3. https://investor.cariboubio.com/news-releases/news-release-details/caribou-biosciences-reports-second-quarter-2023-financial
  4. 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
  5. https://investors.vrtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-announce-licensing-agreement
  6. https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-and-shoreline-biosciences-enter-definitive
  7. https://ir.intelliatx.com/news-releases/news-release-details/intellia-and-onk-therapeutics-announce-collaboration-advance
  8. https://investors.primemedicine.com/news-releases/news-release-details/prime-medicine-and-cimeio-therapeutics-announce-research

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