When a pharmaceutical company launches a new drug or starts a new business, it is important to analyze the target market from the perspective of market formation. A detailed study is required to determine whether the introduction of a new product will lead to an increase in market share in the existing market, or whether it will open up new needs and expand the size of the market itself. In this presentation, our principal consultant in Japan will explain the concept of market formation from the perspective of how to understand market competition.

Adragos is a newly created CDMO which – since the very beginning – included digitalization as a key pillar of its business strategy. At the core of the digitalization strategy are customer centricity and decision-making support.

Adragos initiated a corporate program which will cover its manufacturing network leveraging data and digitalization of processes. As part of this program, Adragos is implementing a platform to interact with customers and to support daily operations and decision making.

Drug shortage has continued for two years, mainly on generic medicines, causing confusion in the medical field. And there is still no solution for it, in sight. This time, I would like to consider its true cause and the concept of measures to solve it, and, in relation to this, like to talk about thinking way of how to secure a stable supply of API and raw materials under global risks. 

Since 2020, there have been repeated cases of voluntary recalls and administrative penalties due to GMP violations at pharmaceutical manufacturing facilities. I would like to summarize what happened in these cases and why they could not be avoided, and consider what pharmaceutical manufacturing facilities should do.

Cancer has a diverse molecular background, including types of genetic mutations, which results in different mode of progression, available molecular targeted therapies, and their efficacy from individual to individual. Since clinical application of liquid biopsy technologies to diagnose the molecular biological information that defines such diversity for personalized cancer care is accelerating, here I would like to share some examples and future prospects.

Under the initiatives of accelerating translational research, it has become possible to carry out drug development widely in academia from "manufacturing", non-clinical trials, to investigator-initiated clinical trials. On the other hand, the essential role of academia in drug creation, such as basic research such as elucidation of disease pathology and exploratory investigator-initiated clinical trials, is also becoming important. Open innovation across interdisciplinary fields such as chemistry, biology, and information engineering is essential.

The importance of manufacturing biologics is increasing recently. In this presentation, we will introduce the activities of the MAB organization toward therapeutic antibody and gene-therapy vector manufacturing.

The manufacturing of drug substances for clinical trials of peptides corresponds to the initial stage of development in the product life cycle. It must be based on thorough GMP control. We will explain the points to note in global GMP control when manufacturing clinical trial peptide drug substances.

Regenerative medicine products have a wide range of raw materials (cell sources), properties, and handling methods. As a result, the approach in process design and examination is often on a case-by-case basis.
Solid risk management is essential for proper manufacturing, and this seminar will introduce the importance of such risk management.

In the first half, we will introduce biopharmaceutical characterization workflow and analytical methods including multidimensional HPLC.
In the second half, we will talk about automation of sample preparation. MAM and LC/MS data analysis, and digital transformation of lab.

In the growing pharmaceutical CDMO market, what is needed for a win-win situation for both contract manufacturers and contract manufacturers? This presentation will explain with the latest case studies and will also focus on sales strategies and human resource development.

Proteins, which are one of main modality, are linear polypeptides of 20 naturally occurring amino acids, then form three-dimensional structures for biological function. The diversity of the amino acid sequences (sequence space) generates various biological functions, but the diversity size is much larger than the size that can be screened experimentally. To explore and find a novel protein sequence in this vast space, unprecedented and phenomenal ways are required. Recently, machine learning has been combined with experimental molecular evolution. The experimental data of molecular evolution is used to train machine learning, and the trained machine guide us to a specific sequence space where functional proteins are enriched. aiProtein^® technology, which is the machine-learning-based AI-assisted protein engineering technology developed by RevolKa, efficiently explores sequence space with small numbers of experimental data and enables sequence optimization for multiple protein properties simultaneously. The use of small data, instead of big data, in aiProtein^® saves time and labor, reduces liability, and opens opportunities for complex biological data. In this seminar, we talk about the current situation and future of machine learning application to protein engineering, together introducing aiProtein® technology.

Recently, three different types of human mesenchymal stem cells have been approved as regenerative medicines for cell therapy. However, because it has becoming evident that their conditioned medium has similar therapeutic effects as well, it has attracted much interest as a safer cell-free therapy. In this lecture, we will introduce potential for therapeutic application of the conditioned medium of stem cells from human exfoliated deciduous teeth as a regenerative medicine by immortalizing the stem cells. 

The creation of antibodies that can potentially be used as drugs is extremely important in the development of Biopharmaceuticals,  but it is still difficult to produce antibodies with the desired functionality. We are developing technologies to create functional antibodies based on epitopes to which antibodies bind. In this presentation, we will introduce the platform technologies for selecting the antibodies with superior functions and show some examples of their application.

Background: Plasma microRNA143-3p (miR143) increase in the acute phase of acute myocardial infarction (AMI) was significantly correlated with left ventricular functional recovery in chronic phase.

This finding gave us an opportunity to investigate whether miR-143#12, a synthesized chemically-modified miR-143 derivative, delivers beneficial effects on AMI. Results: In AMI models, 9 μg/kg of miR143#12 significantly reduced the infarct size and significantly improved the cardiac function both in rats and rabbits at 2 weeks. In rats, the miR143#12 increased the number of CD31-positive and α-smooth muscle actin-positive vessels, and of Ki67-positive cardiomyocytes. Electron microscopy revealed that miR-143#12 prevented the autophagic cell death of cardiomyocytes. AMI decreased the content of miR143 in infarct areas, and in situ hybridization revealed the presence of miR143 in the infarct areas 24 hours after the treatment with miR143#12.

In cultured rat H9C2 cells, miR143#12 significantly inhibited the autophagic cell death induced by H2O2, via decreasing ROS level, and increased viable cells number compared with control by silencing COX-1, -2, and ATG7. The neoangiogenesis by miR143#12 in vivo experiment reflected increase in the number of tube formation in human umbilial endothelial cells. Conclusion: MiR143#12 is a new promising strategy for treatment of AMI through silencing plural key genes.

The ratio of biosimilars to marketed biopharmaceuticals in Japan is low, and the replacement of original biopharmaceuticals with biosimilars is not progressing.

The government is promoting support for the development of biosimilars as a measure to reduce medical costs, but on the other hand, it is also taking measures to approve “bio-same” and stifle the development of biosimilars. Are biosimilars unnecessary in Japan? We would like to reconsider what should be done from the standpoints of both companies and patients.

What you develop the robust purification processes by combining some chromatography media effectively has been one of the key factors to achieve the projects in success at the manufacturing scale for the biotherapeutic drugs.
In this presentation, we would like to introduce you some case studies and our Bio-Rad’s process chromatography solutions.

Minaris Regenerative Medicine is a contract development and manufacturing organization (CDMO) for regenerative medicine products with manufacturing sites in Japan, the U.S., and Europe, and offers process development, clinical and commercial manufacturing for cell and gene therapy products.
In the presentation, we will introduce global trends in the regenerative medicine (cell and gene therapy) industry, current challenges and our business.

In the development of therapeutic antibody, advanced antibody engineering technology has enabled the development of antibodies with innovative and unique mechanisms of action, represented by the bispecific antibody. In this presentation, we will introduce the contribution to drug discovery and pharmaceutical technology by the bispecific antibody purification process using the newly developed Protein L resin.

①Oligonucleotide therapeutics, such as antisense oligonucleotide and siRNA, are a promising class of drugs that can modulate the expression levels of target RNA. In addition, they can be produced by chemical synthesis, enabling rapid candidate discovery. In this symposium, I would like to introduce the basics of oligonucleotide therapeutics especially focusing on antisense oligonucleotide.

②Luxna Biotech is a biotech company that aims at clinical application of antisense drugs. We have established drug discovery platform, Luxna’s XNA incorporated Antisense Platform, LuxiAP^TM, consisting of artificial nucleic acids invented by Osaka University. Luxna Biotech is conducting joint drug discovery with pharmaceutical companies, technology licensing, and in-house drug development. In this seminar, I will discuss research results for linking university-developed technologies to drug discovery and share the possibilities of this field.

Pharmaceuticals are being developed in shorter development periods to achieve early approval and long-term profitability, and lower costs are also required. Flow technology is expected to be one of the solutions. This presentation will introduce what is expected of this technology from the viewpoint of pharmaceutical CMC development, as well as examples of research and manufacturing using our flow technology.

Though many case studies of process chemistry have been discussed so far, few ones of research management can be found in process development of drug substances. 
Based on Astellas’s case, I will try to discuss a research management in the process development in this lecture.

In order to provide our customers with even better products, we pursue to “Pure Chemistry,” = “make clean (synthesis),” “purify cleanly (refining),” and “provide clean (analysis)” in process design. We have been striving to create products based on the concept of "Pure Chemistry". This time, we would like tointroduce our process design, focusing on the part of “make clean(flow reaction)”.

Previously, small molecules played the leading role in chemically synthesized pharmaceuticals. However, we are now entering into a new era of multi-modality, where middle to large molecules such as oligonucleotides or peptides and chemically modified biopharmaceuticals such as ADC are quickly on the rise in the pharmaceutical industry. Our examples of pharmaceutical process development and strategy in such an era will be presented.

Many enantiopure drugs contain a hydrogen atom at the stereoselective center. As this hydrogen atom can be introduced into appropriate unsaturated precursors by hydrogenation, asymmetric hydrogenation is of particular importance.

In recent years, with the progress of basic research in the life science field, substances and drug targets that have not been used as drugs in the past are now being utilized. These are attracting attention as a new drug discovery modality. This presentation will focus on oligonucleotide therapeutics among the new drug discovery modalities, and in addition, will give an outlook on future pharmaceuticals.

In this presentation, I would like to introduce the construction of a small molecule compound library based on rigid cage-like scaffolds, which allows substituents to be precisely arranged three-dimensionally with respect to the target protein, to achieve structural diversity in the compound library. Furthermore, I would also like to present the "SAR Matrix" and "SAR Transfer" methods for prediction of side chain structures using ChEMBL databases.

In　the early CNS drug development stages, it is necessary to estimate the extent to which candidate drugs reach the human brain. We have been developing human blood-brain barrier models in order to evaluate drug penetration property to the brain. In this presentation, we will introduce the current status and future perspectives of the BBB models.

Recently biologics such as antibody and gene therapy have gained much attention, but are small molecule drugs completely obsolete? Well, the future of small molecule drugs may not be so gloomy - Evaluate will share where  small molecule drugs sit in the pharmaceutical industry now and in the near future using various data such as our "Consensus Sales Forecast.”

Antibody drugs have developed rapidly since 2000 and have grown to more than half of the biopharmaceutical market in Japan. However, the rise of next-generation antibodies is expected amid the need for various further improvements. In this lecture, I will introduce the prospects for the use of single-domain antibody molecules ticketed in camelids as candidates for next-generation antibodies in pharmaceuticals.

Have you looked beyond CSR in your quest to achieve sustainability? Intangible assets are swiftly gaining recognition as a primary source of competitive advantage for companies, with key advantages derived from human capital, intellectual property and predictions of future competitiveness. In this seminar, EY Japan’s CPAs and sustainability experts will guide participants through a discussion of key concepts pharmaceutical companies should incorporate into their sustainability programs.

In this seminar, I will explain about an open research publication model, F1000, which we describe as "Beyond Open Access". F1000 publication model is supporting many global funders and institutions including Bill & Melinda Gates Foundation. I’ll explore the publication process that ensures transparency of peer review process and quality of papers, while at the same time enables speedy publication as required during pandemic.

As various issues regarding the stable supply of pharmaceuticals and active pharmaceutical ingredients are being questioned, the partnership between Japan and India in the supply chain is now attracting attention. A special seminar by the India Group, which will be held on the first day of CPHI Japan 2023, will examine the challenges and possibilities of the India-Japan partnership, where further mutual cooperation is expected in the pharmaceutical and healthcare industries.