"The vast majority of the thousands of monogenic genetic diseases that have been discovered in the world today have no effective treatment." Boya's founder Wei Wensheng said, "After the emergence of genetic editing technology, theoretically many disease treatments have become feasible. ."
Behind the "feasibility" means countless hopes.
Among the common monogenic genetic diseases, for example, thalassemia, a disease commonly seen in China's Guangdong and Guangxi regions, is dragging down countless families mentally and economically.
In March 2016, China published the “Blue Book of Thalassemia in Chinaâ€. According to relevant data, there are about 300,000 people with severe and intermediate thalassemia in China, and about 30 million people with “thalassaemia†genes. Among them, the provinces and cities such as Guangdong, Guangxi and other provinces have more than 10% of the "thalassaemia" gene carriers, and even more than 20% in Guangxi. These patients usually need to lose red blood cells every month, but this will lead to the deposition of iron in the organs, so you need to take the iron drugs on time and in accordance with the doctor's advice, the treatment cost can reach 100,000 yuan / year, and the only cure It is the transplantation of heterologous hematopoietic stem cells that have been successfully matched. Heterologous hematopoietic stem cells have very high requirements for matching, so the success rate of matching is very low. Most patients can only consume a short life while waiting for a match.
Using genetic editing techniques will make it possible to provide more efficient, safe, and precise treatment options for thalassaemia treatment.
Founded in 2015, Boya is working on the technology transformation of the technology in treatment and drug development. In the words of Wei Wensheng, taking a disease as an entry point, what the company does is a brand new field and the beginning of a revolutionary opportunity in the future.
A few days ago, Boyaa series just announced the completion of the 100 million Pre-B round of financing, led by Lilly Asia Ventures, Huagai Capital and investment, the company A round of investment in the IDG Capital, China Economic Cooperation and Gong Hongjia Wait for investors to continue to follow the investment. In the office building of the Boya Collection in Beijing Changping Life Science Park, the founder of the company Wei Wensheng and CEO Wei Dong accepted an interview with Hunting.com.
Wei Dong revealed to Huntun.com that the funds will be mainly used to promote treatment projects and high-throughput technology research and development and team expansion. The team will include more than 20 people in bioinformatics, new technology development and clinical research.
"Newcomer" CEO: For the opportunity, but also for the challenge
For the Boya series, the big thing that happened "just" is not only this one, Wei Dong itself is also a "big thing."
He graduated from the Department of Biology of Peking University with a Ph.D. from Michigan State University and an MBA from Wharton School. Wei Dong is a professional manager with rich experience in biomedical field. He has led more than ten new drugs. The R&D project has gone through many processes from target search, program design to IND, clinical development, and drug registration.
Last month, he just returned from the United States to join Boya Collection and served as CEO. Wei Wensheng started his jokes in a familiar way: "It's still a newcomer." Wei Dong also ridiculed himself: "Although looking old, it is indeed a newcomer."
The two have a deep relationship. In the early years, Wei Wensheng and Wei Dong were undergraduate students in the Peking University Department of Biology. Later, they went to the University of Michigan State University to study in Bo, and later arrived in the San Francisco Bay Area. However, the former went to Stanford to continue studying, and the latter chose to enter the company. In 2007, Wei Wensheng chose to go back to Peking University to continue his research. Wei Dong has completed the MBA to go on the management road. Wei Wensheng described this as "gradually getting farther away."
Why the reunion happened again, and Wei Wensheng smiled and said that he had taken care of Wei Dong’s “Mao†and attracted three people to laugh.
“I used to work in big companies. Big companies are very strong in terms of capital and technical strength, but they chose to join startups because they have their own biggest impact here.†Wei Dong said, himself I like to study innovative technologies, and the ultimate goal of technology is to bring benefits to patients and society. The Boya series has provided such a platform that it can do what it has been trying to do for the past two or three decades, and it is back. To China, opportunities are also challenges.
More importantly, in Wei Dong's view, whether it is the United States, Europe or China, any small company that ultimately wants to succeed must have two characteristics. First, science and technology are first-class, and second, the team is first-class. “Although the Boya series is small, it is difficult to find such a powerful platform in China, including the world.â€
Wei Wensheng founded the Boya series in 2015, mainly after he applied for a patent for the invention of new technology at Peking University Laboratories, and needed a strong commercial platform. “Translating the latest technology born in the laboratory into therapeutic and drug research and development services.†Wei Dong said that this is the biggest advantage of the Boya series. At the same time, the 52-person team covered 9 doctors, 6 returnees, and 27 graduates with master's degree or above.
"Four platforms" to overcome the challenges of treatment and drug development
According to public information, the world's first successful case of gene therapy occurred in the United States in 1990. Due to the lack of adenosine deaminase in the body, a 4 year old and 9 year old girl suffered from severe combined immunodeficiency, scientists. The first use of gene therapy has been successful.
“Especially after the invention of CRISPR technology in 2012, gene editing technology has been widely used, from agriculture, animal husbandry, to human medical technology research, and now medical treatment, there is a very big prospect.†Wei Dong told Hunting cloud network. As a powerful "genome editing" technology, CRISPR can trim, cut, replace or add biological DNA sequences.
The Boya series focuses on the treatment of human diseases and drug development, and based on this, has created three gene editing cell therapy platforms and a high-throughput genetic screening platform.
Among them, the hematopoietic stem cell treatment platform is dedicated to the study of blood-borne genetic diseases. The first project of Boya is the thalassemia mentioned at the beginning of this article.
The treatment of “thalassaemia†is mainly based on hematopoietic stem cell transplantation, because the process requires the use of hematopoietic stem cells from others, which requires prior matching, which is also a difficult point of treatment. "We extracted the hematopoietic stem cells of the thalassemia patients and repaired them and then returned them. In theory, there is no immune rejection problem." Wei Dongru described this treatment process.
In addition to thalassaemia, the hematopoietic stem cell platform is also conducting research and treatment of other blood diseases. Among them, Boya Collection and Tianjin Institute of Blood Research have cooperated with the gene editing and treatment project for the angiocytic anemia.
The second platform is T cell gene editing, which is mainly for tumor diseases. Wei Dong describes the process of its implementation: adding a specially synthesized compass that can bring T cells to specific targets in a T cell. After identifying the target, T cells are activated and mobilize other immune cells together to the cancer cells. Strike.
In 2017, the US FDA (US Food and Drug Administration) approved the listing of Novartis and Kate's CAR-T drugs, which further enhanced confidence in cancer treatment worldwide. "Including domestic, everyone thinks this is a very good way to treat cancer, especially advanced cancer. Chinese companies have also actively researched and developed in this area."
However, Wei Dong mentioned that in fact, including the Novartis and Kate, as well as many Chinese companies in the research and development of the use of autologous cells, the cancer patients themselves T cells are extracted, in vitro transformation processing, and then returned The patient kills the tumor cells. One of the benefits is that it does not cause immune rejection. However, when the patient has undergone repeated chemotherapy or the condition deteriorates, his or her own T cell status will be poor and cannot be effectively modified and processed in vitro. In addition, it takes several weeks from the extraction of patient cells to the preparation of qualified CAR-T cells, and many patients simply can't wait for that moment.
"When we looked at Novartis's approval of the materials, we found that about 15% of the patients died because the autologous cells were not made, or the patients couldn't wait." Wei Dong said, therefore, the Boya series edited the T cell. Emphasis is placed on the "universal variant" CAR-T approach. T cells were extracted from healthy people, and the immune rejection problem was removed by gene editing. The samples were prepared according to different targets and placed in reserve. "In this way, the patient can be used immediately after diagnosis, and its efficacy and quality will be more uniform, so that doctors can control the dosage." In addition, he believes that from the manufacturing point of view, its production efficiency is higher than that of autologous CAR-T. It will also be significantly improved, and mass-produced finished products can eventually be used by hundreds of patients, and the cost will be greatly reduced.
The third cell platform is an in vivo therapy, mainly for genetic diseases of solid organs. Through special carriers, such as viruses and biochemical particles processed in vitro, gene editing tools or some other functional nucleic acid fragments can be reached to target organs for treatment. effect. Wei Dong said with excitement that some of the company's latest technologies are expected to break through the limitations of the CRISPR approach.
The high-throughput genetic screening platform based on CRISPR technology is one of the competitiveness of Boya series, which plays an important role in accelerating drug research and development.
“High-throughput genetic screening technology is still a very new approach compared to traditional drug target screening technology.†Wei Dong said that most companies on the market still use traditional methods, and such as Boya series can use CRISPR technology. There are still very few companies that achieve very high throughput and saturation screening, so their high-throughput genetic screening platforms have the potential to help big pharmaceutical companies and research institutions make breakthroughs.
“Our technology accumulation in high-throughput genetic screening is more than most companies that provide this service. Our improvement in screening efficiency is also more than ten times higher than other companies.†Wei Wensheng added, “But technology The lead is always time-sensitive, because you are chasing after you in the industry."
Clear value on the way to technology
Yong mother is questioned, market prospects, is one of the characteristics of the biomedical industry, the qualities that make the world a number of companies fought to invest a lot of manpower, material and financial resources. However, the long-term development cycle and slow output of the industry itself are still prominently reflected in the development process of biotechnology companies.
In 2016, the world's genetic editing industry ushered in the first time. Three genetic editing companies based on CRISPR technology: Editas Medicine, Intellia Therapeutics, and CRISPR Therapeutics were listed separately. However, in terms of technology, they all encountered bottlenecks in the advancement of clinical trials and even postponed them. The course of clinical trials. Among them, Intellia Therapeutics has not yet announced a clinical trial plan, and Editas Medicine announced that clinical trials of CRISPR-based treatment for a rare hereditary blindness have also been postponed.
“There is a saying in our industry that 20% of the results are Science and 80% are Industrialization.†Wei Dong said.
“CRISPR Therapeutics is the fastest in the three,†he told Hunt Cloud.
At the end of last year, the company Therapeutics announced an application to the European Union government, hoping to use CRISPR to genetically modify the blood cells of patients with beta thalassemia. At present, the approval has been approved, and the first phase of clinical trials is being carried out in Europe. This project is also similar to the first project of Burson-Marsteller, thalassemia. Wei Dong is optimistic about this, which has brought a good opportunity for the company. Since then, there are examples of treatment experience.
For the Boya series of such start-up biotech companies, it is clear that it is not limited to technical experience. How to get investors' favor in the capital market that focuses on financial investment, in order to obtain sufficient funds and time to focus on research, also need to learn from experience.
Wei Wensheng is not particularly worried about this issue. He believes that from the perspective of biotech companies, the way to measure whether it is worth investing is different from the current common measure of profitability. Every time a biotech company advances, there is data showing how likely it is to get to the end. This is a way of valuation. “For example, in our poverty-stricken projects, there will be data in pre-clinical and different clinical stages, and the possibility of final success can be assessed in real time.†More importantly, “we are different from developing Internet technology products. Internet technology products company Can be developed but I don't know if anyone wants it, and the 'users' of biomedical products are definitely wanting but the company may not be able to develop them."
The prospect is broad, and the user “needs†is one aspect, but the application of genetic editing technology itself is not long, but it takes 5 to 7 years. Lack of professional knowledge, it is easy for ordinary people to be wary and suspicious of new technologies.
Wei Dong thinks that this is a normal phenomenon. He recalls a problem in his strategic counseling: every time a new technology comes, everyone will have high expectations, and then expect a collapse, and finally return to a reasonable expectation. “This is a very typical adoption curve.†He believes that genetic editing is more accurate than traditional techniques, but the current stage is not 100% accurate, which is why it is clinical. Previously, it was necessary to repeatedly evaluate the reasons for its safety.
More importantly, "in the case of not being able to prove its negative nature, you can only use the data to speak last, but you can't go ahead because there are some unpredictable risks."
The significance of moving forward lies in innovation and independent research and development.
Wei Wensheng defines what the Boya series has done as "from 0 to 1." In his view, genetic editing techniques may be more likely to have milestone development outcomes than other biotechnologies. There is a difference between technology and technology. Compared with the original technology, the rise of gene editing technology is short-lived, and the future is full of hope and unknown. Choosing this road means that you need to “cross the river by feeling the stonesâ€, which is very challenging for Boya.
"We are doing something new and of great value." He said that this is the risk that the Boya series can bear, and it can be "excitingly exciting."
The so-called veterinary drugs, referred to as veterinary drugs, refer to drugs that can regulate the function of livestock from the body and prevent and cure livestock and poultry diseases. Natural plants, animals, minerals, and synthetic drugs and immunizations can all be used as veterinary drugs. Feed additives used in animal production are also often included in the category of veterinary medicines
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