Catalyst Awardee

Project Description

Development of nucleic acid therapeutics with built-in DDS

YOSHIOKA Kotaro, PhD, MD | Tokyo Medical and Dental University;
Competition Sponsor: 
Japan Agency for Medical Research and Development
Awardee Year: 2023

Nucleic acid therapeutics are rapidly advancing as a new class of molecular-targeting drugs following antibody therapies. However, two main challenges remain: inadequate drug delivery to target tissues and risk of nucleic acid-induced toxicity. To overcome these challenges, Dr. Kotaro Yoshioka has developed a novel double-stranded oligonucleotide technology, overhanging heteroduplex oligonucleotide (ODO) that enables inherent nucleic acid-based drug delivery system (DDS). The ODO technology enables much more efficient drug delivery to target tissues and higher safety compared to conventional nucleic acid therapies, resulting in an expansion of the therapeutic window by more than 10-fold.
Leveraging rapid development of nucleic acid therapeutics for clinical application, this innovation is able to make rapid contributions toward extending healthy lifespan through groundbreaking curative treatments for debilitating diseases. In addition, the versatility of this ODO-technology, applicable to diverse effects such as gene expression suppression or upregulation and splicing regulation, holds promise for addressing a wide range of challenging pathology. Consequently, this ODO-technology provide the substantial potential for societal contributions to various diseases, spanning from life-threatening diseases such as cancer to neurodegenerative disorders like Alzheimer’s disease and mental health conditions like depression.
In this project, we aim to elucidate the correlation between nucleic acid molecular structure and interdisciplinary characteristics through the utilization of novel molecular techniques, including nanoscale nucleic acid chemical modification design and atomic-level analysis of target molecules. Subsequently, development of DDS-intrinsic nucleic acid molecules tailored to individual target organs will be undertaken in a progressive and novel manner.

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