The Rare Disease Consortium Japan (RDCJ) is a co-creation platform founded on the philosophy of “leaving no one behind” and dedicated to advancing drug development for rare diseases. It brings together industry, patients and patient groups, academia, government, and citizens to connect the entire pathway from basic research to clinical application and social implementation. By overcoming institutional and sectoral boundaries, RDCJ accelerates consensus-building and aligns development with the patient’s timeline. The approval of viltolarsen, an exon-skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy, demonstrated that Japan can establish rare disease drug development processes consistent with global standards. Moreover, the expansion of exon skipping illustrates how a foundation built for one disease can be extended to other rare diseases and therapeutic modalities. As a shared platform, RDCJ promotes standardisation through annual meetings and working groups, leverages cross-disease infrastructure, and links Japan’s innovations to the world─delivering new treatments to patients without delay.

This article describes my research stay at the Gonçalo Bernardes Lab, University of Cambridge. I participated in drug discovery projects, including the development of small-molecule RNA degraders and drug conjugates, in collaboration with academic and industry partners. The lab environment encouraged researchers to lead projects independently and engage with a diverse range of techniques. Interactions with industry highlighted the importance of concise communication and clear decision-making. Through my research life in Cambridge, I was able to explore the challenges and possibilities of translational chemical biology. I hope this report will be helpful to those considering international research experiences.

Medicinal chemistry has evolved from its foundations in organic synthesis into an interdisciplinary science, now integrating chemical biology and diverse therapeutic modalities. The development of innovative medicines increasingly requires collaborative drug discovery, where academia, industry, and biotech ventures combine their strengths. The COVID-19 response exemplified this, as diverse foundational technologies converged through collaborative efforts to deliver vaccines and therapeutics in a remarkably short time. Such advances underscore the need for diverse expertise and perspectives, including the active participation of women researchers. Globally, efforts to highlight and support women in medicinal chemistry are expanding, yet in Japan women still account for only 18.5% of all researchers, indicating substantial untapped potential. This special issue, Women Shaping the Future of Medicinal Chemistry, presents the challenges and achievements of leading women researchers in Japan, offering a vision of how diversity is shaping the future of the field.

Natural products are compounds produced by living organisms, and have been developed into many medicines. Aiming to isolate natural products using protein binding ability as an indicator, we have designed a “target protein oriented natural product isolation method” using protein beads. We obtained compounds that binds to the transcription factor hairy and enhancer of split 1 (Hes1), which suppress neural differentiation, and activate neural stem cells to differentiate into neurons. We also isolated and determined the structure of Notch signal inhibitors to activate differentiation of neural stem cells. We also developed a co-culture method of microorganisms and animal cells to activate silent genes in microorganisms. Pathogenic microorganisms produce compounds that suppress immune activity to survive against immune cells. We also analyzed the interactions between microorganisms and immune cells. In this paper, we introduce examples of our research in natural product chemical biology.

Photoimmunotherapy is a novel cancer treatment in which an antibody conjugated with the near-infrared dye IR700 is administered to the patient. After administration, the target site is irradiated with light, triggering a photochemical reaction that selectively damages targeted cancer cell membrane. The author had the opportunity to be involved in its discovery at the U.S. National Institutes of Health (NIH) as a postdoc. After returning to Japan, the mechanism by which photoexcitation induces axial ligand cleavage and aggregation of IR700, initiating cell damage, was elucidated through collaborative research. These findings are now being utilized to design more effective therapeutic agents. As there are many challenges that cannot be addressed alone, research is routinely advanced in collaboration with other researchers. Laboratory staff and students are also essential members. With the support of many people, the author intends to continue efforts to refine photoimmunotherapy and expand its applications.

After my research experience in the laboratory of Prof. Schreiber, my main research field has become medicinal chemistry and chemical biology. For the purpose of functional regulation of histone methyltransferases using compounds, we are trying to develop novel drug candidates including both inhibitors and activators. This time, I described the research background related to development of novel modulators of a histone H3 lysine 27 (H3K27) methyltransferase complex, and also mentioned an overview of activator development. Furthermore, a story of laboratory management as a new PI and the prospect for development of innovative drug candidates are indicated. Through the chemical epigenetics research, I will continue with my efforts to obtain novel chemical modulators by developing a research strategy in this field.

I took my first step as a researcher at Osaka University Graduate School of Pharmaceutical Sciences, where I focused on DDS (Drug Delivery System) research, particularly in biopharmaceutical DDS. After graduating, I learned about drug manufacturability and quality control in the CMC division of Daiichi Sankyo Co., Ltd. Eventually, I transitioned to the RD department, where I have been involved in modality drug discovery utilizing DDS technologies up to the present. Throughout this journey, I have worked on developing antibody drug enhancement techniques and LNP technologies, and most recently, I had the opportunity to participate in the development of a COVID-19 vaccine. In this article, I will share the beliefs I developed during my graduate studies as a researcher and the lessons I learned from my experience in COVID-19 vaccine development.

This article presents a case study from Takeda’s global COVID-19 drug discovery initiative, demonstrating how diverse approaches and creative perspectives were effectively applied during the lead generation stage. By collaborating across Takeda research sites and external partners, the team leveraged computational chemistry to design non-covalent inhibitors targeting conserved regions of the coronavirus (CoV) main proteases (Mpro). This strategy enabled the discovery of lead compounds with pan-coronavirus Mpro inhibitory activity across multiple CoV strains. The project exemplifies how structural insights and scientific innovation intersect with strategic team management. Behind the creative output was an environment shaped by thoughtful leadership─respecting individual strengths, assigning roles accordingly, and guiding decision-making through scientific evidence. The findings highlight that team diversity, combined with adaptive and data-driven leadership, plays a critical role in accelerating innovation under urgent global conditions.

Chemical probes are small molecules that selectively interact with specific biomolecules to modulate or detect their functions. Since chemical biology itself is a relatively new interdisciplinary field, creatively utilizing these probes in the drug discovery process can play a significant role in developing innovative pharmaceuticals. As this essay collection focuses on female researchers pioneering medicinal chemistry research, I would like to introduce Otsuka Pharmaceutical’s journey in applying chemical biology research to drug discovery, alongside my own personal career development experiences.