Led by Associate Professor Hiroya Kuwahara of Department of Neurology and Neurological Science and the Nucleotide and Peptide Drug Discovery (TIDE) Center at the Institute of Science Tokyo,
"Advancing personalized nucleic acid therapeutics for rare and intractable diseases" has been selected as a new research project for fiscal year 2026 under the "Research Project for Practical Application of Intractable Diseases" by the Japan Agency for Medical Research and Development (AMED).
In recent years, dramatic advances in genome analysis technology have enabled the identification of genetic abnormalities found in only a very small number of patients. However, conventional industry-led drug development is not economically viable for such cases, and the lack of therapeutic development for these patients has emerged as a significant unmet medical need.
This research aims to implement, for the first case in Japan, a new drug development paradigm known as "personalized (N-of-1+) drug development", in which nucleic acid therapeutics that act directly on the genetic abnormalities are developed for a single patient or a very small number of patients. In particular, we aim to establish a robust international collaboration with the n-Lorem Foundation in the United States-a global pioneer in "personalized (N-of-1+) drug development" using antisense oligonucleotides, the most actively developed class of nucleic acid therapeutics-and to introduce into Japan the personalized clinical trial currently being implemented in the United States.
Through this research, we anticipate establishing new pathways to therapeutic access for patients with rare diseases, a small patient group worldwide. The collaboration will initially focus on pursuing treatment of a small number of nano-rare patients who reside in Japan.
Project Information
Project Name: Research Project for Practical Application of Intractable Diseases
Call for Proposals: Research Fields Contributing to the Promotion of Personalized Medicine for Rare and Intractable Diseases
Call Category: (E-5) Research on the Promotion of N-of-1+ Drug Discovery (N-of-1+ Drug Discovery)
Research and Development Project Title: Advancing personalized nucleic acid therapeutics for rare and intractable diseases
Research and Development Period
From May 2026 to March 31, 2030 (planned)
Research and Development Team
Principal Investigator:
Hiroya Kuwahara (Associate Professor, Department of Neurology and Neurological Science and the Nucleotide and Peptide Drug Discovery (TIDE) Center at the Institute of Science Tokyo)
Principal Research Collaborators:
Kumiko Ui-Tei (Specially Appointed Professor and Director, the Nucleotide and Peptide Drug Discovery (TIDE) Center at the Institute of Science Tokyo)
Hidehiro Mizusawa (President Emeritus, National Center of Neurology and Psychiatry; Director, Genomic Medicine Japan)
Masatoshi Takagi (Professor, Department of Pediatrics and Developmental Biology, Institute of Science Tokyo)
