[Depth] Six questions about "gene editing" that is the best in China

Recently, Luo Wenbo, chief macro strategist of Zhongtai Securities, released a 35-page research report on "Genetic Editing - God Scissors Ushering in the First Year of the Eruption." In the research report, the main answer is how to quantify the third-generation genetic editing (CRISPR) relative to the first two generations of side effects and cost advantages. What are the application prospects of gene editing and how it differs from tumor cell immunotherapy such as CAR-T? How is the genetic editing market space measured? What are the clear catalysis of gene editing 2016 major trials? What other research and commercialization cases are there in addition to the Yellow Army in China? What is the understanding of the prospects for teamwork between Jinjia and Huang Jun? The six major issues that everyone pays close attention to.

1. How does the third-generation gene editing (CRISPR) quantify the side effects and cost advantages of the previous two generations?

The great improvement in off-target rate, efficiency, portability and cost is a revolutionary feature of CRISPR compared to the previous two generations of gene editing technology. For the quantification of these advantages that investors care about, although the results of different sites are different, the data shows: Compared with the previous two generations, the efficiency of the company has increased by more than 10 times, the ease of improvement has been 100-1000 times, the construction period has been shortened to 1/12, and the cost has been reduced from 5,000 dollars to 30 dollars. The latest technical progress of the off-target rate can be reduced to the current technical test. Not at the level.

2. What are the application prospects of gene editing and what are the similarities and differences with tumor cell immunotherapy such as CAR-T?

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 be divided into in vivo gene editing (for the complete cure of AIDS, hepatitis B and other viral diseases) and in vitro gene editing. Among them, hematopoietic stem cells (ie, induced IPS) for treating genetic diseases such as thalassemia and T cells (ie, TCR or CAR-T) for treating tumors are two of the most imaginative areas of genetic editing in the future.

　　 CRISPR can help breed the next generation of CAR-T therapy and achieve the king of tumor immunotherapy

First of all, CAR-T has significant advantages over other cellular immunotherapy, but high cost, tumorigenicity, and self-supply are difficult to mass-produce are key bottlenecks limiting CAR-T applications.

Secondly, from the perspective of curative effect, the combination of CRISPR and CAR-T is expected to solve the four major bottlenecks that plague CAR-T. First, CRISPR high-efficiency fixed-point insertion of genes is expected to solve the tumorigenic problems caused by random insertion in the past. Second, the use of CRISPR Efficient knockout of immunosuppressive genes such as PD-1 is expected to inhibit the environment and thus solve the problem of poor CAR-T solid tumors. Third, compared with the previous two generations, the cost of editing CRISPR genes has been reduced from US$5,000 to US$30. The US dollar, which reduces the current expensive cost of CAR-T up to 500,000 US dollars; Fourth, the use of CRISPR directed knockout of genes related to immune rejection is expected to achieve allogeneic treatment.

In addition, from the action of the most advanced pharmaceutical companies in the world, foreign CAR-T therapy giants have joined hands with genetic editors, and the combination of CAR-T and CRISPR is the general trend.

Finally, according to the calculation of the Zhongtai Medical Group, after the CAR-T bottleneck is broken, the market size of domestic blood cancer alone will exceed 70 billion yuan.

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