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
Rena have the following three business lines.
This is a business form for licensing out HDO technology.
This is a business form of collaborative R&D with customers, in which Rena design and provide HDO compounds and advance collaborative R&D to find lead HDOs.
The HDO manufacturing business is a business form in which Rena manufactures, supplies and sells HDOs for target genes only for research and development.
For inquiries about the introduction of our technology