Vol.18, March 2020
Shiro Akinaga, Ph.D.
President & CEO
１．Background and Goals of the Company
AccuRna is a biotech company founded in December 2015.
It is based on the DDS technology of the drug delivery system of Kazunori Kataoka, the director of iCONM and is also a professor at the University of Tokyo.
Also, creating ventures companies for social implementation of smart nanomachines is one of the objectives COINS, so AccuRna exists to support such activities.
AccuRna has two main areas of business.
One is drug discovery pipeline, so-called drug discovery. It functions as a mini-pharmaceutical company, but as you all know, in biotech, there is a very high hurdle to receive approval for selling, and manufacturing drugs.
One of the pillars of our business is conducting drug discovery in the early stages and making alliances with pharmaceutical and biotech companies.
Another pillar is giving non-exclusive licenses of our original patented technology to pharmaceutical companies, academia, or biotech to carry out various kinds of incubation.
Basically, AccuRNA targets the short SiRNA type of RNA within nucleic acid medicine. The antisense oligo, a sibling of siRNA, is DNA.
This includes so-called short oligonucleic acids and another, messenger RNA. We use this messenger RNA itself for medicine. The notable aspect about messenger RNA treatment is that it doesn't get into the genome.
Because it is a bar code, it is transcribed once, so in that sense there is merit.
2.Features of the Research and Recent Advantages
siRNAs are unstable, they are not administered directly like antisense oligos. Patisiran, a nucleic acid drug (siRNA), uses Liquid Nano Particles (LNP). This is generally very versatile and is a rival to our polymer.
In fact, the development of siRNA as an anticancer drug using this LNP has not been successful. Therefore, we are also considering conducting new clinical trials. (described later in this video)
The only successful drug delivery system is targeting the liver. But, eventually, targeting only the liver is not enough.
If you find that it is highly effective, you want to do something other than the liver, for example, tumors. Our merit is that YBC polymers are tumor selective and do not go to the liver very much. As for messenger RNA, today's YBC polymers can go anywhere with an intravenous injection.
In fact, we are already preparing for a Investigator Initiated Clinical Trial using our YBC polymer. What we do is to make nucleic acid medicines with GMP compliance and help with formulation. We also support the administrative aspects of Investigator Initiated Clinical Trials.
On December 6, last year, I submitted an application for a clinical trial. The 'first patient in' is expected in February or March 2020.
For about two and a half years, we have invested heavily in this asset. Finally, we have reached a Investigator Initiated Clinical Trial. Professor Kataoka is very pleased that his research will enter clinic trials, and he has high expectations. There are collaborative studies looking at new biomarkers, and we trying to get a lot of information from this trial.
I think there are three main goals for the future. One is to deliver the new siRNA and antisense oligo, that is our YBC polymer, to places other than the liver, mainly to tumors. Now we are focusing breast cancer and glioblastoma. We are trying to fight completely different type of cancers than liver cancers.
It is not yet known whether these two targeted cancers are suitable for YBC polymers.
I think that a partnership for use for other cancers will be necessary to determine and know the strengths of the YBC polymer.
Ultimately, there is a big competitor, Liquid Nano Particles (LNP), which I mentioned earlier.
We want to differentiate from LNP and clarify its strengths. To that end, the first near-future goal is to create the strengths of DDS through clinical research rather than basic research.
The second is the so-called precision medicine approach, which already being perused. It uses messenger RNA (Neo Antigen Vaccine) to read individual patient's entire antigens using Next Generation Sequencing and dropping them into messenger RNA and immunization.
Nucleic acid medicine is a format that it is most suitable for precision medicine. Antibodies can be developed as precision medicine, but antibodies are characterized by a long half-life, so making it into personalized medicine is technically difficult at this stage. This is the second point that nucleic acid medicine can be taken it to personalized medicine.
There is one opportunity for joint research on messenger RNA research with Professor Idaka at Tokyo Medical and Dental University.
There are two methods: using the messenger RNA directly as a drug, or knocking down the messenger RNA with an antisense oligo or siRNA. Our appeal forms the first slide of our recent corporate pitch called “Messenger RNA as a target for therapy”.
4．Expectations for King Skyfront
About the benefits of entering iCONM. Professor Kataoka has provided his patents and know-how. We are conducting collaborative research while maintaining close communication with him.
Without contact, humans do not know each other, and nothing is created where there is no communication. Being close is a great advantage. The good news is that Haneda Airport beyond this river is surprisingly distant now, but a bridge will soon be completed over the river, so I have great expectations for King Skyfront.