1、 Technical introduction

(1)Structure of HDO

HDO is composed as follows.

Active strand(DNA) : Function as medicinal effect
Carrier strand(RNA) : Function as transportation of active strand (medicinal effect) to disease site
Ligand : By binding a substance that specifically binds to a specific receptor to the carrier strand, it functions as a transport to a targeted disease site

(2)The mechanism of action of HDO

The carrier strand (RNA) of HDO incorporated into the cell is cleaved by RNase H (enzyme).

The active strand (DNA) remaining after cleavage binds to the target mRNA.

When the active strand (DNA) binds to mRNA, it becomes DNA / RNA structure again, and RNase H cleaves the target mRNA.

As mRNA is cleaved, expression of mRNA is suppressed, and drug efficacy is demonstrated.

※RNase H has the property of cleaving RNA among double strands composed of DNA / RNA.

(3)Strengths of HDO technology

HDO has the following strengths.

  • By linking the ligand to the carrier strand, it is possible to selectively deliver the active strand to the diseased site, so that high efficacy can be obtained.
  • Since HDO adopts a double-stranded structure, HDO is essentially stable, it is possible to avoid toxicity by avoiding chemical modification conventionally applied to the active strand which is a medicinal part
  • Since HDO has a double-stranded structure, it becomes a materially stable structure and the enzyme resistance in blood improves
  • Since a ligand is bound to a carrier strand having a transporting function, there is a low possibility that an unexpected medicinal effect is caused depending on a ligand (if the ligand is bound to an active strand, the medicinal effect may be remarkably lowered)
  • There is no need to make the linker used for binding the ligand to the carrier strand dare to break easily
  • The position and the number of ligands bound to the carrier strand can be freely selected

2、 Business Introduction

(1)Business model

We are aiming to create new nucleic acid drugs through platform type business model.

It is not a business model (so-called pipeline model) that derives development medicine that we undertook drug research and development to pharmaceutical companies etc. and obtains consideration.
From the stage of drug discovery research, we are collaborating with pharmaceutical companies and developing business models that develop drugs together with pharmaceutical companies and others.
By doing so, we are aiming for early commercialization and monetization while controlling investment funds.

(2)Pillars of business

We are engaged in 1) joint Collaborative search business, 2) basic technology licensing business, and 3) nucleic acid drug development business.

  1. Collaborative Search : It is a business to create developed drugs with pharmaceutical companies and others.
  2. Basic technology licensing business :  We are licensing pharmaceutical companies and others to license HDO basic technology patents etc.
  3. Nucleic acid drug development business : It is a business to license out development drug created in-house.


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