Science and Technology Platform Program for Advanced Biological Medicine

Management system

Expectations for the “Science and Technology Platform Program for Advanced Biological Medicine”
Proposals for diverse research and development themes selected with the aim of accumulating expertise in 5 years

Toshio Miyata, Program Supervisor

Drug development is a good example of a knowledge-intensive industry being focused on by Japan and is expected to be a growing next-generation industry. Recently introduced policies for accelerating translational research have enabled academic researchers to carry out exploration for hit compounds, structural optimization, synthesis and formulation of medicines under Good Manufacturing Practice, nonclinical studies under Good Laboratory Practice, and investigator-initiated clinical studies. As a result, expertise in these processes is steadily accumulating for small molecule drugs. However, this is not the case with biological medicines. Maintaining the quality and consistency of biological medicines is difficult because the conditions for cell culture, purification, and concentration need to be strictly controlled. Even though there are novel academic findings, there are many hurdles to clear before clinical application. Many pharmaceutical companies have not yet accumulated enough experience and expertise to commercialize biological medicines. At present, academia, contract research organizations, and pharmaceutical companies (biotech ventures) are independently gaining experience in the development of biological medicines, and there is not yet adequate cooperation and collaboration among them.

To develop innovative biological medicines in Japan, the discovery of component technologies and concepts itself is not enough. Combining component technologies can create added value and increase the likelihood of clinical applications. Crucial to these efforts is an open innovation platform where academia, CRO, and pharmaceutical companies (biotech ventures) can share their experience and expertise, and work together to identify and solve various problems during development of biological medicines. Without such activities, innovative biological medicines will not be realized despite innovative academic findings because there will still be unsolved problems regarding production technology, intellectual property, and regulatory affairs. We have to gain experience and expertise specific to the development of biological medicine.

Japan Agency for medical Research and Development (AMED) ran the Basic Science and Platform Technology Program for Innovative Biological Medicine for 5 years beginning in 2014. Out-licensing was a program goal. A system providing necessary support for intellectual properties, contracts, non-clinical studies, and regulatory affairs was established within the program. The program was successful, producing 40 authorized out-licensing, 61 patent applications (40 PCT applications), and 8 startups.

The successor of that program is the “Science and Technology Platform Program for Advanced Biological Medicine”, started in 2019, which will run f or 5 years. Twenty-seven proposals on diverse research themes (antibody medicine, peptide medicine, nucleic acid medicine, cell therapy, gene therapy, genome editing, and delivery system) were selected for the program based on their high potential for development of innovative biological medicines, and I am looking forward to seeing the progress of these projects. A support team comprising experts at out-licensing, intellectual property, startups, and development of biological medicines was established within the program. We will run the program in a cooperative and flexible manner in order to achieve fruitful outcomes.

Advanced biological medicines from Japan to the world

Yasushi Kaneda, Program Officer

My specialty is gene therapy. Several gene therapies were approved in recent years, mainly in Europe and the United States, and they are attracting attention as powerful therapies for genetic disorders and cancer. However, gene therapy used to be an abandoned area because early gene therapy was generally unsuccessful and caused serious adverse effects. It was only after 2011 that high efficacy was shown thank to steady progress in basic research. In Japan, the clinical study of gene therapy was started at Hokkaido University in 1995, but the first approval for clinical use was only this year (HGF gene for peripheral vascular disease). Why did it take so long? Many problems have been pointed out. Firstly, most researchers were unaware of the significance of translational research and clinical studies necessary for approval, the need for startups, and strategies for intellectual property. This was gradually addressed by the program for translational research established by Ministry of Education, Culture, Sports, Science and Technology, Japan (MEXT) in 2007. Meanwhile, the United States and China are dominating the world’s research through massive investment, and there is a fear that Japan is losing ground. However, given the high level of research originality in Japan, the development of innovative medicines here is very much possible through the establishment of the system to identify promising research projects and provide adequate funding and personnel for those projects, and through reform of regulations to allow rapid commercialization of research findings. The “Science and Technology Platform Program for Advanced Biological Medicine” carefully selected research projects that will produce advanced technologies for innovative next-generation medicine, provides MEXT funds, and aims for out-licensing of technologies within 5 years with the help by an experienced support team. As a program officer, I am happy about our selected projects. I would like Japanese pharmaceutical companies to show their determination to lead the world with Japanese technologies: this program will surely produce worthwhile technologies for this goal.

Continuous development and out-licensing of biological medicines in an all-Japan framework

Yoshihiro Otaki, Program Officer

In recent years in the life sciences, whole-genome analysis data generated by next-generation sequencing are accumulating and innovative research tools have been established, such as iPS cells and genome editing. There are high hopes that use of these technologies will enable the development of new medicines with novel structures or novel mechanisms, and we need to be globally competitive in conducting research and development in this area. As part of the international research community, Japan has consistently contributed to basic research (e.g., discovery of targets). Japan’s translational activities have lagged behind those in Europe and the United States and thus are not highly regarded. For the continuous introduction of biological medicines developed in Japan, it is essential to establish a system facilitating swift commercialization of innovative technologies, in addition to discovering the technologies themselves. In other words, intellectual property management and project management for achieving this goal within the given schedule are required along with the systematic accumulation of evidence. This program aims at out-licensing of research findings within 5 years for rapid commercialization. More precisely, the program offers a strong support system for each step in the development of biological medicines, ranging from basic research to commercialization: such support is not adequate within academia. In this program, researchers will collaborate closely with the support team/members to achieve continuous development of biological medicine technologies and their out-licensing in an all-Japan framework.

Clear criteria have been set for authorization of out-licensing, and each case will be reviewed at an authorization meeting.

Other programs related to the development of medicines are underway in parallel at the Japanese Agency for Medical Research and Development. Organic inter-program collaboration will be beneficial for the development of world-leading biological medicines.

Out-licensing of research findings is a primary program goal

Tadashi Horiuchi, Program Officer

The “Basic Science and Platform Technology Program for Innovative Biological Medicine” was started by Japan Agency for Medical Research and Development (AMED) in 2014 with the aim of out-licensing Japanese technologies within 5 years. This hugely successful program, which produced out-licensing of 40 technologies, was widely praised, thanks to tremendous efforts by principal investigators, and support by program supervisors, program officers and intellectual property officers. The main primary of the successor program “Science and Technology Platform Program for Advanced Biological Medicine”, started in 2019, is also out-licensing of technologies to industry. I moved to Keio University Hospital Clinical and Translational Research Center to be involved in translational research after a long career in research and development at a pharmaceutical company. I maintain good relationships with researchers in the pharmaceutical and diagnostic industries and related sectors, and I hope to utilize my broad network for out-licensing of technologies developed through this new 5-year program. I look forward to your support and cooperation in developing world-leading biological medicines in this program.

We must combine a “bold strategy” and “sophisticated tactics”

Kouhei Tsumoto, Program Officer

When I first started my research on the engineering of antibody proteins about 25 years ago, I could not imagine the situation today. Many Japanese researchers have been successful in advancing component technologies, but the key in the development of medicines is combining component technologies. This means that the research and development of a medicine is now difficult for a research group or even a company to do alone. This is why we must now combine a “bold strategy” and “sophisticated tactics”.

In the past 5 years, the number of antibody medicines (including the next-generation antibody medicines) has neared 100, nucleic acid medicines have become realistic therapeutic options, and expectations for cell therapy (e.g., CART therapy) and gene therapy have risen. Research and development of these modalities require interdisciplinary research, as well as advancement of component technologies: refinement of both strategy and tactics is essential to achieve the ultimate goal—the development of medicines.

Such a requirement was met by including out-licensing as a new evaluation criterion in the “Basic Science and Platform Technology Program for Innovative Biological Medicine”. For example, researchers who had been renowned for their basic research were able to improve their reputations internationally through the program, and at the same time, made great achievements in “out-licensing” and “start-ups” that garnered praised.

The successor program, the “Science and Technology Platform Program for Advanced Biological Medicine”, also values interdisciplinary research. Key elements of this program are the development of powerful technologies suitable for developing next-generation biological medicines and the development of basic science and platform technologies for highly promising modalities (i.e., gene therapy and cell therapy).

Both domestically and internationally, Japan was seen as falling behind in the research and development of biological medicines. However, our “sophisticated tactics” are steadily producing good results. I will spare no effort in supporting this program and contributing to the implementation of our “bold strategy”.

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