글로벌 유전자 편집 도구 시장 – 2023-2030

Overview
Global gene editing tools market reached US$ 5.8 billion in 2022 and is expected to reach US$ 22.0 billion by 2030 growing with a CAGR of 18.7% during the forecast period 2023-2030.
CRISPR Therapeutics, and Vertex Pharmaceuticals, are part of the Kelly CRISPR & Gene Editing Technology ETF and are expected to launch the first-ever U.S.-marketed treatment based on CRISPR this year. The treatment, called Exa-cel, is a gene-editing cure for sickle cell disease and beta-thalassemia and could become the first-ever CRISPR-based medicine to hit the market.
The FDA has granted the drug a priority review for sickle cell disease, with a decision expected by December 8, 2023. If approved, this would be a significant milestone in the Life Sciences Industrial Revolution, which could set expectations for future gene-editing programs. Vertex is leading the global development, manufacturing, and commercialization of Exa-cel and is splitting program costs and profits worldwide 60/40 with CRISPR Therapeutics.
Gene editing tools are revolutionary technologies that empower scientists to precisely modify the genetic material of living organisms. These tools, which include CRISPR-Cas9, TALENs, and ZFNs, allow researchers to insert, delete, or alter specific DNA sequences with an unprecedented level of accuracy.
This breakthrough has far-reaching implications across various fields such as medicine, agriculture, and biotechnology, as it allows for the development of targeted therapies, the creation of genetically modified organisms, and the advancement of scientific knowledge. Gene editing tools hold immense potential to address genetic diseases, enhance crop yields, and drive innovations that shape the future of genetics and personalized medicine.
Furthermore, the rapid advancement and adoption of gene editing tools across various industries, and rising investment by the market players are the factors expected to drive the market over the forecast period.
Dynamics
Increasing Research in Developing in vivo RNA-based Gene Editing Model for Blood Disorders is Expected to Drive the Market Growth
On July 27, 2023, a team of researchers at the Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania successfully created a model for administering gene editing tools directly to the body to treat blood disorders. This development could pave the way for the direct modification of diseased blood cells in the body, leading to a significant breakthrough in the treatment of blood disorders.
In their experiments, the researchers used CRISPR-based gene editing tools to modify the genes of blood cells in mice, and the results were promising. This breakthrough is particularly significant because it may make ex vivo gene editing unnecessary. This process requires removing cells from the body, editing them in a lab, and then returning them to the body. In vivo gene editing, on the other hand, is a potentially more efficient and less invasive approach to treating blood disorders.
Hence, owing to the above mentioned factors, the market is expected to drive during the forecast period.
Increasing Clinical Results of Allogeneic CRISPR-Based CAR-T Therapy Targeting CD19 with PD-1 Knockout for Aggressive Non-Hodgkin's Lymphomas is Expected to Drive the Market Growth.
In December 2022, Caribou Biosciences released positive results from their trial in the US of an allogeneic CRISPR-based CAR-T therapy that targets CD19 in aggressive non-Hodgkin's lymphomas that are difficult to treat. The therapy also features a second genetic modification that disables the PD-1 gene, which cancer cells sometimes use to evade the immune system. The treatment was generally well-tolerated and had an acceptable safety profile.
In a phase 1 trial with 6 participants, all of them initially showed total remission. Two of the participants had no signs of the disease a year later and are still being monitored by the trial. Caribou Biosciences' product has been granted RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designations by the FDA, based on this initial data. This is a groundbreaking development in the field of gene editing and cancer treatment.
The therapy's success in targeting CD19 and deactivating PD-1 has demonstrated promising safety and efficacy results in a phase 1 trial for non-Hodgkin's lymphomas. The ability to modify genes using CRISPR technology opens up new possibilities for personalized and targeted cancer therapies. More research and clinical trials will be required to confirm these findings and determine the long-term effectiveness of this approach.
The field of gene editing is constantly advancing and this groundbreaking therapy has the potential to transform cancer care with personalized and targeted treatments. Further research and clinical trials will be crucial in establishing the long-term effectiveness of this therapy and ushering in a new era of precision medicine for cancer patients.
Regulatory Challenges in Gene Editing in Orphan Crops are Expected to Hamper the Market Growth.
Gene editing in crops is subject to varying regulatory environments across countries and regions, which can hinder the adoption and implementation of gene editing technologies in orphan crops. Certain orphan crops may face additional regulatory obstacles due to their unique genetic makeups or limited commercial value.
For instance, the European Union (EU) has a stringent approach to regulating gene-edited crops and food, effectively prohibiting their introduction. In contrast, the United States (US) has a more lenient regulatory framework. Such differences pose challenges for companies and researchers operating across different regions.
The EU's strict regulations stem from the European Court of Justice's (ECJ) 2018 ruling that gene editing must be regulated under the 2001 GMO Directive, which restricts genetically modified crops that use genes from another species (e.g., transgenic crops). Thus, gene-edited crops and food are banned in the EU.
However, in September 2022, EU agriculture ministers discussed revising the rules on gene editing, acknowledging the benefits of precision techniques that enable easier and faster modification of a plant's DNA (cisgenics). Therefore, the varying regulatory frameworks across different countries and regions, such as the EU and the US, can pose challenges to the widespread adoption and implementation of gene editing technologies in crops. Streamlining regulations through harmonization can aid in the efficient development and commercialization of gene-edited crops, making them more accessible and adopted globally.
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Segment Analysis
The global gene editing tools market is segmented based on type, application end-user, and region.
Owing to the Precision and Versatility, Cost-Effectiveness, Rapid Development, Broad Applicability, Innovation, and Investment, the Type Segment Accounted for Approximately XX% of the Gene Editing Tools Market Share
CRISPR-Cas9, which stands for "Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated protein 9," is a revolutionary gene editing technology that has transformed genetic research and applications. Originally discovered as a natural defense mechanism in bacteria against viral infections, scientists harnessed this system to create a powerful tool for precisely modifying DNA sequences in various organisms.
CRISPR-Cas9's ease of use, affordability, and applicability across a wide range of organisms have democratized gene editing, allowing researchers to tackle questions that were once challenging or impossible to address. While its potential is immense, ethical considerations and the need for precision and safety continue to be important aspects of its utilization.
Furthermore, on September 16, 2022, Intellia Therapeutics, Inc., a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, announced positive interim results from an ongoing Phase 1/2 clinical study of NTLA-2002, its second in vivo genome editing candidate.
NTLA-2002 is a systemically administered CRISPR candidate being developed for hereditary angioedema (HAE) and is designed to knock out the KLKB1 gene in liver cells, thereby reducing the production of kallikrein protein. The uncontrolled activity of kallikrein is responsible for the overproduction of bradykinin, which leads to the recurring, debilitating, and potentially fatal swelling attacks that occur in people living with HAE. The interim data were shared today in an oral presentation at the 2022 Bradykinin Symposium held in Berlin, Germany.
Additionally, on June 14, 2022, the abstract was presented at the European Hematology Association (EHA) Congress, a group of researchers that includes Children’s Hospital of Philadelphia (CHOP) presented new data on an investigational therapy for transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD). The one-time treatment, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, showed continued benefits at up to three years after administration, with a safety profile as expected for autologous transplant and potentially much safer than allogeneic transplant (from a donor).
The abstract provides new data from two clinical trials on exa-cel (exagamglogene autotemcel), formerly known as CTX001, a one-time treatment that utilizes CRISPR gene editing to boost the production of fetal hemoglobin to correct the defective gene for hemoglobin associated with both diseases. Hence, owing to the above factors, the market segment is expected to hold the largest market share over the forecast period.
Geographical Penetration
North America Accounted for Approximately 43.7% of the Market Share in 2022, Owing to the Investing in Gene-Editing Stocks, Partnerships among Market Players, and the Developement of a New Gene-Editing System
North America is expected to hold the largest market share over the forecast period. Owing to the development of new gene-editing systems, increasing investment in gene-editing stocks, and partnerships among market players.
For instance, in February 2023, Moderna announced a partnership with ElevateBio to expand its gene editing research. The collaboration will utilize Life Edit's proprietary gene editing technologies, such as base editing, along with Moderna's mRNA platform to create potentially permanent treatments for rare genetic diseases and other conditions.
Base editing is a tool that uses CRISPR technology to modify a single base in the DNA code. Moderna will provide funding for preclinical research studies conducted by the two companies, using Life Edit's CRISPR-based tools that enable precise modifications to the human genome. The partnership's objective is to develop innovative gene therapies for the future. This joint venture highlights the potential for advancements in gene-editing therapies and ongoing efforts in the field of gene editing.
Hence, owing to the above factors, the North America region is expected to hold the largest market share over the forecast period.
Competitive Landscape
The major global players in the market include CRISPR Therapeutics, Intellia Therapeutics, Precision BioSciences, Synthego, Sangamo Therapeutics, Cellectis, Precigen, GenScript, Editas Medicine, and Caribou Biosciences among others.
COVID-19 Impact Analysis
COVID-19 has moderately impacted the gene editing tools market. The urgency to understand and combat the virus led to rapid research and development. Gene editing tools played a crucial role in studying the virus's genetics, creating diagnostic tests, and investigating potential therapies. mRNA-based platforms, a type of gene editing technology, played a pivotal role in developing COVID-19 vaccines like Pfizer-BioNTech and Moderna.
This highlighted the potential of these tools in responding to emergent infectious diseases. Researchers repurposed existing gene editing techniques to develop diagnostic tests for detecting SARS-CoV-2, demonstrating the adaptability and versatility of these tools.
However, shutdowns and restrictions had an impact on laboratory work and gene editing research, particularly in non-COVID-related areas. This delay slowed down progress in various projects. Resources were redirected to COVID-19-related research, which strained funding and attention. This could potentially limit investments and advancements in gene editing projects unrelated to the pandemic.
Clinical trials for gene therapies, which often require rigorous testing, were affected by disruptions in patient recruitment, monitoring, and regulatory processes. Lockdowns also hindered collaboration and communication among researchers, delaying information sharing and slowing down the dissemination of new findings.
Russia Ukraine Conflict Analysis
The ongoing war between Russia and Ukraine may have negative effects on scientific research and technology, particularly on gene editing tools. If research institutions, universities, or laboratories in either country are impacted by the conflict, it could disrupt ongoing scientific research, including gene editing studies. The movement of researchers, availability of resources, and communication between international collaborators may be hindered, which could slow down the progress of gene editing projects.
During times of conflict, governments tend to prioritize funding for defense and security, which could result in reduced investment in other areas like scientific research. This may limit the availability of resources for gene editing research, including funding for equipment, facilities, and personnel.
Additionally, international collaboration is crucial to the advancement of gene editing tools, but the conflict could make it more challenging for researchers from different countries, including Russia and Ukraine, to collaborate effectively due to travel restrictions, political tensions, and communication challenges.
By Type
• CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated protein 9)
• Transcription Activator-Like Effector Nucleases (TALENs)
• Zinc Finger Nucleases (ZFNs)
By Application
• Medical Therapies and Treatments
• Drug Development
• Agriculture and Food Production
• Biological Research
• Synthetic Biology
• Others
By End User
• Biotech and Pharma Companies
• Academic and Research Institutes
• Contract Research Organization
• Others
By Region
• North America
o U.S.
o Canada
o Mexico
• Europe
o Germany
o U.K.
o France
o Italy
o Spain
o Rest of Europe
• South America
o Brazil
o Argentina
o Rest of South America
• Asia-Pacific
o China
o India
o Japan
o Australia
o Rest of Asia-Pacific
• Middle East and Africa
Key Developments
• On March 21, 2022, Merck KGaA, a leading multinational pharmaceutical company, signed through its Life Science division, MilliporeSigma, a unique collaboration and license agreement with an Israeli AgTech company to demonstrate the utility of its proprietary CRISPR genome-editing tools in agricultural uses. The agreement licenses its foundational CRISPR intellectual property to Israeli BetterSeeds Ltd., a disruptive company that uses genome editing technology including CRISPR to develop new breeds of plants.
• On February 22, 2023, ElevateBio and Moderna joined forces to focus on the development of gene editing therapies using a tool called base editing. This tool can alter a single letter in the DNA code, and the partnership aims to speed up the development of new treatments. To achieve this, Moderna will provide funding for preclinical research led by Life Edit Therapeutics, a subsidiary of ElevateBio based in North Carolina. The research will use CRISPR technologies to create potentially permanent treatments for rare genetic diseases and other conditions. This partnership is part of Moderna's efforts to expand its gene editing portfolio, which uses technologies similar to those employed in its COVID-19 vaccines.
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