개요
세계의 유전자 편집 툴 시장은 2022년에 58억 달러에 달했으며, 2023-2030년의 예측 기간 중 CAGR 18.7%로 성장하며, 2030년에는 220억 달러에 달할 것으로 예상되고 있습니다.
CRISPR 테라퓨틱스와 버텍스 파마슈티컬스(Vertex Pharmaceuticals)는 켈리 CRISPR & 유전자 편집 기술 ETF의 일원으로 올해 미국 최초의 CRISPR 기반 시판용 치료제인 Exa-cel(Exa-cell)을 출시할 예정입니다.에 대한 유전자 편집 치료제로, CRISPR을 이용한 의약품으로는 최초로 시장에 출시될 가능성이 있습니다.
FDA는 겸상적혈구증에 대한 우선 심사를 승인했으며, 2023년 12월 8일까지 결정이 내려질 것으로 예상됩니다. 만약 승인되면 생명과학 산업혁명의 중요한 이정표가 될 것이며, 향후 유전자 편집 프로그램에 대한 기대감을 갖게 될 것입니다. 버텍스는 Exa-cell의 개발, 제조 및 상용화를 주도하고 있으며, 프로그램 비용과 이익을 CRISPR Therapeutics와 60/40으로 세계에서 공유하고 있습니다.
유전자 편집 툴은 과학자들에게 생물의 유전 물질을 정확하게 수정할 수 있는 능력을 부여하는 획기적인 기술로, CRISPR-Cas9, TALEN, ZFN을 포함한 이 툴들은 연구자들이 특정 DNA 서열을 전례 없는 수준으로 정확하게 삽입, 삭제, 수정할 수 있게 해줍니다.
이 획기적인 기술은 의료, 농업, 생명공학 등 다양한 분야에 영향을 미쳐 표적 치료법 개발, 유전자 변형 생물체 제작, 과학적 지식의 향상을 가능하게 합니다. 유전자 편집 툴은 유전성 질환을 해결하고, 작물 수확량을 향상시키며, 유전학 및 맞춤형 의료의 미래를 형성하는 혁신을 촉진할 수 있는 엄청난 잠재력을 가지고 있습니다.
또한 다양한 산업 분야에서 유전자 편집 툴의 급속한 발전과 채택, 그리고 시장 기업의 투자 증가는 예측 기간 중 시장을 주도할 것으로 예상되는 요인입니다.
다이나믹스
혈액 질환에 대한 in vivo RNA 기반 유전자 편집 모델 개발 연구 증가가 시장 성장을 촉진할 것으로 예상됩니다.
2023년 7월 27일, 필라델피아 아동병원(CHOP)과 펜실베이니아대학교 페렐만 의과대학 연구팀은 혈액 질환 치료를 위해 유전자 편집 툴을 체내에 직접 투여하는 모델을 만드는 데 성공했습니다. 이 개발은 체내의 병적 혈액 세포를 직접 수정할 수 있는 길을 열어 혈액 질환 치료에 큰 돌파구를 마련할 수 있을 것으로 보입니다.
연구진은 실험에서 CRISPR 기반 유전자 편집 툴을 사용하여 생쥐의 혈액 세포의 유전자를 변형시켰으며, 그 결과는 매우 유망했습니다. 이 획기적인 성과는 생체 외 유전자 편집이 불필요할 수 있다는 점에서 특히 중요합니다. 이 과정은 세포를 체외로 꺼내어 실험실에서 편집한 후 다시 체내로 되돌려 넣어야 합니다. 반면, 생체내 유전자 편집은 혈액 질환을 치료하는 데 있으며, 보다 효율적이고 덜 침습적인 접근 방식이 될 수 있습니다.
따라서 위의 요인으로 인해 예측 기간 중 시장은 확대될 것으로 예상됩니다.
PD-1 녹아웃에 의한 악성 비호지킨림프종에 대한 CD19를 표적으로 하는 동종 CRISPR 기반 CAR-T 치료제의 임상 성과가 증가함에 따라 시장 성장이 촉진될 것으로 예상됩니다.
지난해 12월, 캐리부 바이오사이언스(Caribou Biosciences)는 치료하기 어려운 침습성 비호지킨림프종 CD19를 표적으로 하는 동종 CRISPR 기반 CAR-T 치료제의 미국 임상시험에서 좋은 결과를 발표했습니다. 이 치료법은 암세포가 면역체계를 회피하기 위해 사용하는 PD-1 유전자를 비활성화하는 두 번째 유전자 변형을 특징으로 합니다. 이 치료법은 대체로 잘 견디는 것으로 나타났으며, 안전성 프로파일도 양호한 것으로 나타났습니다.
6명의 참가자를 대상으로 한 임상 1상 시험에서 참가자 전원이 완전 관해에 도달했습니다. 참가자 중 2명은 1년이 지난 후에도 질병의 징후가 없었으며, 현재도 임상시험을 통해 모니터링하고 있습니다. 이 초기 데이터를 바탕으로 칼리부 바이오사이언스의 제품은 FDA로부터 RMAT(재생의료 첨단치료제) 및 패스트트랙 지정을 받았습니다. 이는 유전자 편집 및 암 치료 분야의 획기적인 발전입니다.
CD19를 표적으로 삼아 PD-1을 불활성화하는 이 치료법은 비호지킨림프종 환자를 대상으로 한 1상 임상시험에서 유망한 안전성 및 유효성 결과를 보여주었으며, CRISPR 기술을 이용한 유전자 변형 능력은 맞춤형 암 치료와 표적 암 치료의 새로운 가능성을 열어줄 것입니다. 새로운 가능성을 열어줍니다. 이러한 결과를 확인하고 이 접근법의 장기적인 효과를 판단하기 위해서는 추가 연구와 임상시험이 필요합니다.
유전자교정 분야는 나날이 발전하고 있으며, 이 획기적인 치료법은 맞춤형 표적 치료를 통해 암 치료를 변화시킬 수 있는 잠재력을 가지고 있습니다. 이 치료법의 장기적인 효과를 입증하고 암 환자를 위한 정밀의료의 새로운 시대를 열기 위해서는 더 많은 연구와 임상시험이 필수적입니다.
희귀작물의 유전자 편집에 대한 규제 문제가 시장 성장에 걸림돌이 될 것으로 예상됩니다.
농작물의 유전자 편집은 국가와 지역에 따라 규제 환경이 다르기 때문에 희귀 작물의 유전자 편집 기술 채택과 도입에 걸림돌이 될 수 있습니다. 일부 희귀 작물은 독특한 유전자 구성과 제한된 상업적 가치로 인해 추가적인 규제 장벽에 직면할 수 있습니다.
예를 들어 유럽연합(EU)은 유전자 편집 작물 및 식품을 규제하는 데 있으며, 엄격한 접근 방식을 취해 사실상 도입을 금지하고 있습니다. 반면 미국은 규제 프레임워크가 느슨한 임베디드니다. 이러한 차이는 각기 기타 지역에서 사업을 운영하는 기업과 연구자들에게 도전이 될 수 있습니다.
EU의 엄격한 규제는 유럽사법재판소(ECJ)가 2018년 유전자 편집은 2001년 유전자변형작물 지침에 따라 규제되어야 한다는 판결에서 비롯된 것으로, 이 지침은 다른 종의 유전자를 사용하는 유전자변형작물(예: 형질전환 작물)을 제한하고 있습니다. 따라서 유전자 편집 작물이나 식품은 EU에서 금지되어 있습니다.
그러나 2022년 9월 EU 농무장관들은 식물의 DNA를 보다 쉽고 빠르게 변형시킬 수 있는 정밀기술(cisgenics)의 장점을 인정하고 유전자 편집에 관한 규정 개정을 논의했습니다. 따라서 EU, 미국 등 국가와 지역별로 상이한 규제 프레임워크는 작물에 대한 유전자 편집 기술의 보급과 도입에 어려움을 초래할 수 있습니다. 규제 조정을 통한 규제 간소화는 유전자 편집 작물의 효율적인 개발 및 상용화를 돕고, 더 쉽게 접근하고 세계에서 채택될 수 있도록 할 수 있습니다.
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.
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.
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.
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.
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.
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.
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 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.
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.
The global gene editing tools market report would provide approximately 61 tables, 61 figures, and 195 Pages.
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