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source:未知 author:admin Release time:2020-02-15 11:56 Browse volume:
Brokerage survey] 6 questions about "gene editing" that are most popular in China
2016-01-22


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On January 15, 2016, Luo Wenbo, chief macrostrategic analyst of Zhongtai Securities, published a 35-page survey report on "Gene Editing Topics-God Scissors Welcomes the First Year of Outbreak". The research report mainly answers how the third-generation gene editing (CRISPR) has quantified side effects and cost advantages compared to the previous two generations? The application prospects of gene editing and how are they similar to tumor cell immunotherapy such as CAR-T? How to measure the gene editing market space? What are the clear catalysts for major experiments in gene editing in 2016? In addition to Huang Jun's team in China, what other scientific research and commercialization cases are there? What do you think about the prospects of team cooperation between Jinjia and Huang Jun? Six issues that everyone is paying close attention to.


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1
How is the third-generation gene editing (CRISPR) advantage compared to the previous two generations in terms of side effects and cost?


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The great improvement in off-target rate, efficiency, portability, and cost are the revolutionary features of CRISPR over the previous two generations of gene editing technology. For the quantification of these advantages that investors care about, although the results are different at different sites, the data shows: Compared with the previous two generations of technology, CRISPR has improved its efficiency by more than 10 times, increased its ease by 100-1000 times, shortened the construction cycle to 1/12, reduced the cost from 5,000 US dollars to 30 US dollars, and the latest off-target technology progress has been reduced to current technology detection. Less than level.


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2
The application prospects of gene editing and how are they similar to tumor cell immunotherapy such as CAR-T?


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Gene editing can be divided into animal and plant breeding and human clinical treatment. Among them, in the field of human clinical treatment, it can also be divided into in vivo gene editing (for completely curing viral diseases such as AIDS and hepatitis B) and in vitro gene editing. Among them, hematopoietic stem cells (ie, inducing IPS) for treating genetic diseases such as thalassemia and T cells (ie, TCR or CAR-T) for treating tumors are the two major gene editing subfields in the future.


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CRISPR could help conceive next-generation CAR-T therapy and become king of tumor immunotherapy


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First of all, CAR-T has significant advantages over other cellular immunotherapy, but high costs, tumorigenicity, and difficulty in mass production of autologous supply are the key bottlenecks that limit the application of CAR-T.


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Secondly, from the perspective of efficacy, the combination of CRISPR and CAR-T is expected to solve the four major bottlenecks that plague CAR-T. First, CRISPR's efficient site-specific insertion of genes is expected to solve the problem of tumorigenicity caused by random insertion in the past; CRISPR efficiently knocks out immunosuppressive genes such as PD-1, which is expected to restrain the environment and solve the problem of poor CAR-T solid tumors. Third, compared to the previous two generations of technology, the cost of CRISPR gene editing has been reduced from $ 5,000 to 30 U.S. dollars, thereby reducing the current cost of up to 500,000 US dollars for CAR-T. Fourth, the use of CRISPR to knock out genes related to immune rejection is expected to achieve allogeneic therapy.


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Furthermore, judging from the actions of the most advanced pharmaceutical companies in the world, foreign CAR-T therapy giants have joined hands with gene editing rookies. The combination of CAR-T and CRISPR is the general trend.


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Finally, according to the calculation of the Chinese and Thai Medicine Group, after the CAR-T bottleneck is broken, the domestic blood cancer market alone will exceed 70 billion yuan.


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3
How to measure the gene editing market space?


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1) In the field of plants and animals, China ’s gene editing and breeding are at the world-leading level, and some varieties have begun to be commercialized. This includes plants: 400 kilograms per acre of “Hua Big Millet”, and animal, the world ’s first gene knockout. In addition to dogs and "Hua Da Mini Longevity Pet Pigs" that sell for 10,000 yuan per head, "not growing up";


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2) In the field of clinical applications, in addition to research, Huang Jun's team is at the world-leading level. The biggest point is that the commercialization of gene editing clinical applications in China is in full swing. This includes two points: first, in terms of total volume At present, more than 50 Chinese research institutions have submitted gene editing patents. In Guangzhou and other regions, dozens of Chinese and even hundreds of research institutions are enthusiastically using third-generation gene editing CRISPR.


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In addition, Jikai Gene, which has just received round B investment from Honghui Capital, has achieved commercial product promotion of third-generation gene editing basic materials such as CRISPR vectors, and is building a cGMP-level genetic engineering virus vector production workshop, or will be The first domestic production workshop built for the industrial application of CAR-T therapy.


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4
What are the clear catalysts for major experiments in gene editing in 2016?


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The analyst analyzed the catalytic effects brought about by the major experiments of gene editing in 2016 from three aspects: basic research on CRISPR, technology itself, clinical and application.


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From the basic research, a series of major key action mechanisms of CRISPR, such as the recognition mechanism of targeted enzymes by targeted enzymes, and targeted cleavage, have been clearly revealed, marking that CRISPR has basically matured and perfected in theory, and has been used for large-scale commercialization in the future. Lay a good foundation for the application;


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The improvement of CRISPR technology itself: the most important breakthrough of the third generation of CRISPR in 2015 lies in the efficiency of the technology itself, especially the off-target rate and off-target rate detection, the "last key bottleneck" that once plagued the large-scale promotion of CRISPR has been overcome in one fell swoop;


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From the perspective of clinical and application areas: Three key areas of CRISPR clinical application in 2015: in vivo editing (gene therapy for viral diseases), induced stem cell IPS (hematological diseases such as thalassemia), and T cells (CAR-T or TCR tumors) Immunotherapy) have made breakthrough progress.


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5
In addition to Huang Jun's team in China, what other scientific research and commercialization cases are there?


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In the field of gene editing clinical treatment, China ’s third-generation gene editing CRISPR is the two most important breakthroughs: 1) Huang Jun has researched the team to modify the HBB gene encoding human β-globin to treat thalassemia. Jia shares to cooperate; 2) At present, as the first echelon of direct commercialization of domestic CRISPR, Gaosheng Biomedicine has cooperated with the Third Military Medical University, Chongqing Medical University, Chinese Academy of Sciences, etc., and is trying to target CRISPR to destroy the hepatitis B virus surface antigen (HBsAg) coding region technology is commercialized to achieve the goal of gene cure for hepatitis B.


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Other major commercial clinical trials to be carried out for the first time in 2016-2017 include Sangamo's gene editing for thalassemia, AIDS, and Editas' first CRISPR trial for congenital cataract.


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Analysts believe that the launch of Editas, founded by Yifeng Feng, a CRISPR founder, as the first and purest third-generation gene editor CRIPSR, may establish an "international valuation anchor" for the gene editing theme, as well as the listing of BGI Valuation at the level of 100 billion yuan will fully catalyze the entire precision medical sector.


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6
What do you think about the prospects of team cooperation between Jinjia and Huang Jun?


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Nature predicts CRISPR for thalassemia as the most important scientific breakthrough in 2016, and analysts at Zhongtai Securities believe that the potential domestic market space in the next five years will exceed 50 billion yuan.


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Regarding the announcement of Jinjia and Huang Jun on the team's cooperation to create the first third-generation gene editing CRISPR pure target of A shares, the analyst emphasized the following points:


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First, Huang Jun has undisputed international leadership in research on CRISPR thalassemia and human clinical applications, and Jinjia cooperated with it to make it a pure third-generation gene editing theme for A shares;


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Second, from the perspective of the operation of the world's most advanced pharmaceutical companies, the application of CRISPR to hematopoietic stem cells and thalassemia is one of the two major commercial applications in the future;


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Third, the application of gene editing to hematopoietic stem cells and thalassemia is the most important technological breakthrough predicted by Nature in 2016. If the experiment is successful, it will bring strong catalysis;


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Fourth, the third generation of gene editing is applied to the thalassaemia market capacity imaging: According to the national severe thalassemia patients at least 15,000 or more, and at an annual rate of 10%, and bone marrow transplant surgery that can be tolerated by thalassemia patients is about 40 Price per million. On this basis, we estimate that the size of the potential domestic market in the next five years is close to 50 billion yuan, even if conservatively estimated at a penetration rate of 1/5, it will bring tens of billions of imagination space.


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Note: This article is extracted from the research report of Zhongtai Securities. The strong position in the article only represents the original analyst's point of view, and the editor has deleted some of these statements.
Link source:
Brokerage survey] 6 questions about "gene editing" that are most popular in China
2016-01-22


Ranch


Ranch


On January 15, 2016, Luo Wenbo, chief macrostrategic analyst of Zhongtai Securities, published a 35-page survey report on "Gene Editing Topics-God Scissors Welcomes the First Year of Outbreak". The research report mainly answers how the third-generation gene editing (CRISPR) has quantified side effects and cost advantages compared to the previous two generations? The application prospects of gene editing and how are they similar to tumor cell immunotherapy such as CAR-T? How to measure the gene editing market space? What are the clear catalysts for major experiments in gene editing in 2016? In addition to Huang Jun's team in China, what other scientific research and commercialization cases are there? What do you think about the prospects of team cooperation between Jinjia and Huang Jun? Six issues that everyone is paying close attention to.


Ranch
1
How is the third-generation gene editing (CRISPR) advantage compared to the previous two generations in terms of side effects and cost?


Ranch


The great improvement in off-target rate, efficiency, portability, and cost are the revolutionary features of CRISPR over the previous two generations of gene editing technology. For the quantification of these advantages that investors care about, although the results are different at different sites, the data shows: Compared with the previous two generations of technology, CRISPR has improved its efficiency by more than 10 times, increased its ease by 100-1000 times, shortened the construction cycle to 1/12, reduced the cost from 5,000 US dollars to 30 US dollars, and the latest off-target technology progress has been reduced to current technology detection. Less than level.


Ranch
2
The application prospects of gene editing and how are they similar to tumor cell immunotherapy such as CAR-T?


Ranch


Ranch


Ranch


Gene editing can be divided into animal and plant breeding and human clinical treatment. Among them, in the field of human clinical treatment, it can also be divided into in vivo gene editing (for completely curing viral diseases such as AIDS and hepatitis B) and in vitro gene editing. Among them, hematopoietic stem cells (ie, inducing IPS) for treating genetic diseases such as thalassemia and T cells (ie, TCR or CAR-T) for treating tumors are the two major gene editing subfields in the future.


Ranch


CRISPR could help conceive next-generation CAR-T therapy and become king of tumor immunotherapy


Ranch


First of all, CAR-T has significant advantages over other cellular immunotherapy, but high costs, tumorigenicity, and difficulty in mass production of autologous supply are the key bottlenecks that limit the application of CAR-T.


Ranch


Secondly, from the perspective of efficacy, the combination of CRISPR and CAR-T is expected to solve the four major bottlenecks that plague CAR-T. First, CRISPR's efficient site-specific insertion of genes is expected to solve the problem of tumorigenicity caused by random insertion in the past; second, the use of CRISPR efficiently knocks out immunosuppressive genes such as PD-1, which is expected to restrain the environment and solve the problem of poor CAR-T solid tumors. Third, compared to the previous two generations of technology, the cost of CRISPR gene editing has been reduced from $ 5,000 to 30 U.S. dollars, thereby reducing the current cost of up to 500,000 US dollars for CAR-T. Fourth, the use of CRISPR to knock out genes related to immune rejection is expected to achieve allogeneic therapy.


Ranch


Furthermore, judging from the actions of the most advanced pharmaceutical companies in the world, foreign CAR-T therapy giants have joined hands with gene editing rookies. The combination of CAR-T and CRISPR is the general trend.


Ranch


Finally, according to the calculation of the Chinese and Thai Medicine Group, after the CAR-T bottleneck is broken, the domestic blood cancer market alone will exceed 70 billion yuan.


Ranch
3
How to measure the gene editing market space?


Ranch


1) In the field of plants and animals, China ’s gene editing and breeding are at the world-leading level, and some varieties have begun to be commercialized. This includes plants: 400 kilograms per acre of “Hua Big Millet”, and animal, the world ’s first gene knockout. In addition to dogs and "Hua Da Mini Longevity Pet Pigs" that sell for 10,000 yuan per head, "not growing up";


Ranch


Ranch


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2) In the field of clinical applications, in addition to research, Huang Jun's team is at the world-leading level. The biggest point is that the commercialization of gene editing clinical applications in China is in full swing. This includes two points: first, in terms of total volume At present, more than 50 Chinese research institutions have submitted gene editing patents. In Guangzhou and other regions, dozens of Chinese and even hundreds of research institutions are enthusiastically using third-generation gene editing CRISPR.


Ranch


In addition, Jikai Gene, which has just received round B investment from Honghui Capital, has achieved commercial product promotion of third-generation gene editing basic materials such as CRISPR vectors, and is building a cGMP-level genetic engineering virus vector production workshop, or will be The first domestic production workshop built for the industrial application of CAR-T therapy.


Ranch
4
What are the clear catalysts for major experiments in gene editing in 2016?


Ranch


The analyst analyzed the catalytic effects brought about by the major experiments of gene editing in 2016 from three aspects: basic research on CRISPR, technology itself, clinical and application.


Ranch


From the basic research: A series of major key action mechanisms of CRISPR, such as the recognition mechanism of targeted enzymes by targeted enzymes, and targeted cleavage, have been clearly revealed, indicating that CRISPR has been theoretically based.

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