"The cell gene therapy industry will definitely have ups and downs in the development process. In recent years, with the in-depth research and development of human genome and the development of medical means, human demand for health level has become more personalized. Cell gene therapy is the latest technology developed under this general trend, and it is a response to our personalized needs. In this way, the global investment in the cell gene therapy industry is very large, and many products have been listed, bringing good news to patients. " He Yingke pointed out that cell gene therapy involves a wide range of technical knowledge, with a wide variety of products and a relatively small scale. Many companies only know a part of the technology. Coupled with complex regulatory requirements, if we want to learn from R&; From ampd to production and listing, there will be a great demand for funds, time and talents to achieve full process coverage. External use of CDMO is a major trend in cell gene therapy industry.
In fact, the data can also confirm the current strong demand in this market. According to Jost Sullivan's analysis, the global cell gene therapy industry began to develop rapidly from 20 15. From 20 16 to 2020, the market scale will increase from 50.4 million US dollars to 2.08 billion US dollars. It is estimated that by 2025, the global cell gene therapy market will reach nearly 30.54 billion US dollars.
Based on the analysis of the cell gene therapy industry, Everbright Securities believes that with the continuous optimization of CDMO technology, the cost of cell gene therapy will decrease rapidly, the accessibility of CGT drugs will increase, and the penetration rate of CGT drugs will further increase rapidly, which is conducive to the rapid development of CGT CDMO. In this way, we are optimistic about companies with unique technical platforms in plasmid, virus and cell production.
CGT is highly dependent on CDMO.
Gene therapy is a new generation of precision therapy after targeted therapy of small molecules and large molecules. It provides a new treatment concept and means for refractory diseases such as tumors, rare diseases and chronic diseases, and has long-term and curative effects that ordinary drugs may not achieve. The huge market potential has promoted the global commercialization of cell gene therapy drugs.
"Since 2020, CDMO for cell and gene therapy has been favored and laid out by capital. However, the core technologies in the upstream of the industrial chain, including reagents and high-end equipment, are still mastered by foreign countries. We believe that localization is an inevitable trend, but it takes time for the underlying technological breakthroughs. " Wu Xiaoying said.
How to break the barriers of CGT industry?
First of all, in terms of virus vectors, the biggest problem is large-scale commercial production. Whether AAV virus or other slow viruses, the production cost is very high. Some raw and auxiliary materials need to be further improved in terms of quality standards, and there are still some imperfect links in production technology. Large-scale commercial development is the next problem to be overcome.
In addition, due to clinical differences, most cell gene therapy projects, whether under research or approved, are still in the field of rare diseases. On the one hand, the widespread social awareness of rare diseases in China needs to be improved, and the financial support needs to be strengthened. On the other hand, the research and development of new drugs, the support of clinical trial foundation and the research of innovative drugs for rare diseases by FDA in the United States have some incentives, including giving priority to the review of new drugs.
When talking about the challenges faced by CGT, Wu Xiaoying also pointed out that there are indeed high technical barriers in the field of cell and gene therapy. We do see that low-level technology, especially high-end equipment and reagents, is also restricted by foreign countries.
He Yingke introduced that from a technical point of view, there are two key points in the formation of industrialization and commercialization: on the one hand, from a biomedical point of view, how to solve problems and treat related diseases? Is the choice of operators good enough? Is it easier to reach more patients and help them faster? This needs to be promoted from the perspective of biomedical basic research, which is why upstream scientific research institutions or biotechnology companies need to develop special technology platforms; On the other hand, from the perspective of industrialization and industrialization, how did CDMO transform these early R&D achievements into industrialization? In this respect, we also need to cooperate with biotech companies like Cytiva in all directions to find breakthrough directions in some industries. For example, how to produce personalized products more automatically through automation technology.
"According to statistics, 70% of the global cell gene therapy companies are still in the preclinical stage, so it is very necessary for CDMO companies with rich industrial experience, technology and production capacity to help them cross the process from preclinical to clinical. Good CMC process will greatly accelerate product development. For the biopharmaceutical industry, the process itself is a product. " He Yingke said.
Related Q&A: What is the meaning of cdmo? Cdmo, namely customized R&D and production in medical field, is a new R&D and production outsourcing model.
Related introduction:
The customization of R&D and the production of CDMO include clinical stage and commercialization stage. Generally, the intermediates needed to synthesize new drugs are provided to domestic and foreign customers from the first or second phase of clinical practice, and then they are deeply bound with customers in the stage of market review and approval and commercial production.
As the front-end extension of CDMO business, technology development services include the discovery and synthesis of new drug compounds, as well as various pre-process research and development services.
Medical CDMO is a deep penetration of the whole industrial chain from preclinical research, clinical trials to product production, providing innovative process research and development and production services for enterprises, and replacing simple capacity output with high value-added technology output.