Boya Group Has Completed The 450 Million Financing Gene Editing Station Of Round B To Accelerate Clinical Transformation

The Nobel Prize for gene editing is still in the air.

On October 13, gene editing company Boya Jiyin announced the completion of 450 million yuan round B financing. Founded in 2015, the company is a biopharmaceutical enterprise based on genome editing technology to accelerate drug research and develop innovative therapies for a variety of diseases. In 2018, the company has established a clinical transformation and application base in Nansha District of Guangzhou in line with GMP standards. In the past two years, Boya Jiyin has completed the financing of RMB 700 million, which will be used to promote the clinical transformation of R & D pipeline and further expand the team.

In an exclusive interview with 21st century economic reporter, Dr. Wei Dong, CEO of Boya Jiyin, said that as a subversive technology, gene editing technology can play a role in a variety of application scenarios, and has great potential in medical application, scientific research, crop improvement, food safety and other aspects. In terms of medicine, gene editing technology also has many transformation directions, including service, diagnosis, treatment and so on.

Wei Dong pointed out to the 21st century economic reporter that in terms of basic scientific research related to gene editing, the number of related papers and patents in China is almost equal to that of the United States. However, it is different at the transformation level, and there is a certain gap in the registered clinical trials, ecosystem and international leading level. "The formation of China's relevant ecosystems requires the joint efforts of enterprises in this field on the one hand, and the support and guidance of national policies on the other hand."

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In recent years, with the development of science and technology, gene editing has become the focus of attention, and the gene editing industry has also ushered in a tuyere.

As a subversive technology, gene editing technology can play a role in a variety of application scenarios. The discovery of CRISPR system has brought a breakthrough in this field. It won the Nobel Prize eight years after its discovery, which shows this point.

Cong Le, the first doctoral student of MIT / Broad Institute Zhang Feng laboratory and now assistant professor of Stanford University Medical School, also told 21st century economic reporter that the recognition of the Nobel Prize for gene editing technology reveals the influence and importance of this technology.

In terms of medicine, Wei Dong said that gene editing technology also has many transformation directions, including service, diagnosis and treatment. Among them, examples of services include high-throughput genome editing and screening, which can provide biological big data support for drug research and development and improve the efficiency of new drug research and development; diagnosis examples include the detection of new coronavirus, which we are most familiar with. This year, the United States urgently authorized a new coronavirus detection kit based on CRISPR / CAS gene editing technology. In terms of treatment, a variety of therapies for genetic diseases and cancer have entered clinical practice, and the treatment of cardiovascular and neurological diseases is expected to enter clinical practice in the near future.

And treatment is the direction that Boya has chosen now.

Wei Dong pointed out that gene editing therapy, which treats diseases from the gene level, is a completely different field from the previous common small molecule and large molecule drugs. "The principle of gene editing therapy is that" treating diseases should be based on the foundation ". From a scientific point of view, some diseases can be cured once and for all."

In the transformation of gene editing technology into therapy, a "toolbox" can be formed according to the characteristics of the disease and the needs of patients, so as to better serve patients. From the point of view of editing objects, it can be divided into DNA Editing and RNA editing. DNA Editing is a permanent change of genetic information, RNA base editing is reversible, and the editing effect is dose-dependent. These two kinds of editing can meet the needs of patients based on their different technologies.

Dr. Wang Minchuan, partner of Sanzheng health investment, pointed out that gene editing, as a revolutionary technology in the field of life medicine, is rapidly promoting its clinical transformation worldwide.

It is understood that at present, boyagin has built a number of research and development platforms, including in vitro cell gene editing and treatment platform for hematopoietic stem cells and T cells, in vivo gene therapy platform based on RNA single base editing technology, and high-throughput genome editing and screening platform dedicated to targeted drug research and development. Among them, CRISPR system, which has just won the Nobel Prize in chemistry, and Leaper, a RNA single base editing system independently developed by Professor Wei Wensheng, founder of Boya science, Peking University.

On the platform of hematopoietic stem cells, there are the fastest-growing projects of Boya Jiyin. Through gene editing, autologous hematopoietic stem cell transplantation is used to treat transfusion dependent beta thalassemia. The nature of the project and its application is based on the characteristics of the patient.

According to incomplete statistics, there are about 300000 severe and intermediate thalassemia patients in China, mainly concentrated in the provinces south of the Yangtze River, especially in Guangdong and Guangdong. There are 16.8% gene carriers in Guangdong and more than 20% in Guangxi. If two carriers get married, the probability of having children with thalassemia is 1 / 4. Patients with severe beta thalassemia usually live less than 20 years without effective treatment.

At present, the treatment of severe β - thalassemia mainly includes regular blood transfusion combined with iron removal therapy and allogeneic hematopoietic stem cell transplantation. Patients with regular blood transfusion and iron removal therapy need lifelong blood transfusion, and 85% of thalassemia patients will encounter severe iron overload, and usually have a short survival time; allogeneic hematopoietic stem cell transplantation matching is difficult, few patients can find the appropriate matching, and the risk of GVHD and immune rejection after treatment is high. For patients with severe beta thalassemia, they are eager for gene editing autologous hematopoietic stem cell transplantation, which is expected to achieve a one-time cure.

On the in vivo gene therapy platform based on RNA single base editing technology, Boya Jiyin applies the RNA single base editing technology Leaper independently developed by Professor Wei Wensheng's laboratory. The application of this technology can realize long-term treatment or one-time cure through different delivery methods according to the needs of patients.

Ecosystem to be built

In fact, because gene editing has a great impact on human life, it has also triggered some ethical discussions. Wei Dong told the 21st century economic reporter that in the international scientific community, there is a clear consensus on the application boundary of gene editing technology, and several top scientists have also jointly signed in the journal Nature: germ cell editing is not allowed in the short term, but somatic cell editing, due to its great potential and application value in treatment We need to accelerate.

According to Cong Le, gene editing is both a challenge and an opportunity. In terms of scientific research support for gene editing technologies such as agriculture and environment, including the exploration of gene editing and treatment for rare and serious diseases, Chinese scientists and some start-up enterprises have taken the lead in the world.

It is reported that since 2013, China's gene editing has taken off, and China has invested a lot in basic research on biotechnology. At present, the number of papers and patents related to gene editing in China is almost equal to that in the United States.

However, Wei Dong pointed out that there is still a big gap between China and foreign countries in terms of transformation.

Wei Dong said that China implements a dual track clinical research system, that is, clinical trials initiated by researchers and registered clinical trials conducted by the drug evaluation center of the State Drug Administration after accepting the application for new drug research (ind).

In terms of clinical trials initiated by researchers, in 2016, China launched the world's first human clinical trial of CRISPR gene editing technology under this framework. In 2019, Chinese scientists completed the world's first preliminary report on the treatment of AIDS and leukemia patients through gene editing stem cells.

However, in the past year and a half, at least 11 gene editing R & D projects have entered the clinical development stage in the United States and the European Union. However, up to now, there has not been a case of gene editing registration clinical trial conducted by the State Drug Administration after accepting the new drug research application (ind) in China.

Registered clinical trials need to make great efforts in pharmaceutical development, preclinical development and clinical program design. From the perspective of the safety of the whole enterprise, it is necessary to understand the safety of various products and technologies from the perspective of science and technology.

"There are also various ICH guidelines. Because at this time, we are making medicine for human body. We need to be responsible for the safety of patients and the quality of products. Therefore, we must do a lot of preclinical experiments before we can enter the preclinical cells and animal models, and see some safety signals. When these are added together, we can understand how to apply them in clinical practice Wei Dong explained.

At the same time, Wei Dong also pointed out that there is still a certain gap between China's gene editing and the international leading level in terms of ecosystem. At present, the United States has a number of gene editing enterprises with a market value of more than US $1 billion, including CRISPR therapeutics, editas medicine, intellia therapeutics, beam therapeutics, etc., which have formed a agglomeration effect in Boston area, and can also make full use of the advantages of the local pharmaceutical innovation ecosystem. The formation of China's relevant ecosystem needs the joint efforts of enterprises in this field on the one hand, and the support and guidance of policies on the other hand.