[17] Prof. Takehiko Wada

Technology

Development of "Next-Generation Nucleic Acid Therapeutics" with No Side Effects, Efficiently Expressing Drug Efficacy in Target Disease Cells
This initiative focuses on developing a molecular foundation for next-generation artificial nucleic acid therapeutics that can recognize the unique intracellular environments of target disease cells, such as those in cancer or stroke. These therapeutics efficiently express drug efficacy exclusively within target disease cells, avoiding side effects in normal cells. Beyond cancer and stroke, nucleic acid therapeutics hold promise for a wide range of applications, including infectious diseases, genetic disorders, and ultra-rare "n-of-1" diseases. As a next-generation modality for molecular-targeted therapies following antibody-based drugs, this platform technology development is highly anticipated

Strength

Development of an Ideal Next-Generation Molecular Targeted Drug, "Nucleic Acid Therapeutics," with High Efficacy in Small Doses and Minimal Side Effects
Nucleic acid therapeutics are emerging as the next-generation molecular targeted drugs expected to address the limitations of antibody drugs, which, despite their high efficacy, face challenges in broader applications. These therapeutics hold promise for treating a wide range of conditions, from genetic disorders like muscular dystrophy to cancers and infectious diseases. The success of mRNA vaccines against COVID-19 has further fueled anticipation for their practical application as mainstream drugs.

  • While nucleic acid therapeutics show significant potential, two major challenges hinder their widespread adoption:
    1. Off-target effects, encompassing side effects caused by binding to RNAs with sequences similar to the target RNA.
    2. Low intracellular permeability, which reduces drug efficacy due to insufficient delivery into cells.
  • To tackle these issues, our research laboratory has developed proprietary technology based on a unique concept: creating drugs that avoid side effects in normal cells while achieving high efficacy with minimal dosage. This innovation led to the successful development of new artificial nucleic acid therapeutics that selectively target diseases such as cancer and stroke, delivering highly effective treatments.
  • Our novel artificial nucleic acid platform is recognized globally for its originality and is considered a groundbreaking strategy for overcoming the fundamental challenges of nucleic acid therapeutics.

(Key Feature of Nucleic Acid Therapeutics 1)
They can be developed and manufactured more cost-effectively than conventional antibody drugs
Nucleic acid therapeutics, which can be produced through chemical synthesis, require lower technological demands for manufacturing compared to antibody drugs, which necessitate advanced biotechnology and stringent product management using cells. This results in reduced facility and raw material costs, enabling relatively inexpensive mass production and lowering barriers to entry. Furthermore, unlike antibody drugs, whose intellectual property is largely dominated by Western countries, nucleic acid therapeutics allow for easier entry not only for pharmaceutical companies but also for industries like chemical manufacturing.

(Key Feature of Nucleic Acid Therapeutics 2)
They offer the potential for the development and production of treatments for rare diseases in the future.
With the dramatic advancements in genetic analysis technologies, identifying the RNA sequences responsible for target diseases and obtaining therapeutic target RNA sequence information has become more time- and cost-efficient. Inspired by the rapid development and high efficacy of COVID-19 mRNA vaccines, nucleic acid therapeutics are gaining attention as an effective strategy for treating a wide range of diseases. Additionally, as exemplified by the approval of nucleic acid therapeutics for muscular dystrophy, these drugs can be precisely designed based on information about the causative gene sequences, even for rare diseases that previously had no treatments. Ultimately, they hold the potential for tailor-made therapeutics tailored to individual patients, revolutionizing drug discovery strategies.
Unlike conventional antibody drugs, which have not been pursued for rare diseases due to profitability concerns relative to development costs.)

Use Case

1. Development of Nucleic Acid Therapeutics That Selectively Target Cancer Cells
Creating safe and highly effective drugs that respond specifically to cancer cell-specific information while avoiding side effects in normal cells

2. Development of Treatments for Generic & Rare DiseasesThe characteristics of nucleic acid therapeutics—allowing for tailor-made treatments for rare diseases at relatively low development costs—make them well-suited for collaboration with developing countries. Developing treatments for rare diseases that have previously been abandoned holds the potential to improve national birth rates and provide greater mental reassurance for people

Message from the Lab

What I want to know

Institute of Multidisciplinary Research for Advanced Materials,
Tohoku University

Professor Takehiko Wada

Why are interested in studying Nucleic Acid Medicine
We are not asking for a business plan, as creating a business idea around nucleic acid medicine can be challenging. If you are interested in nucleic acid medicine, we encourage you to submit your thoughts freely, without being constrained by the requirements of the application task

Desired Candidate Profile

For Those Who Aspire to Bring Happiness to the World and Their Own Country through Pharmaceutical Development
As mentioned, nucleic acid medicine is a new drug discovery with the potential to transform the essence of healthcare and is well-suited for collaboration with developing countries. We aim to equip passionate individuals who wish to tackle significant problems such as improving national birth rates, enhancing people’s quality of life, and fostering a sense of mental security through healthcare. These individuals can then contribute to building a foundation for drug discovery and talent development in their home countries.

Reference