Vol.15, April 2019
Managing Executive Officer, Member of the Board
Division Manager,Regenerative Medicine Division
Background and aims
We opened the facility called Tonomachi CPC in Life Innovation Center for our research and development on regenerative medicine product of Muse cells that we are developing now.
Clinical trial of our Muse Cells was initiated for acute myocardial infarction in January 2018, then cerebral infarction in September and epidermolysis bullosa in December as target diseases.
If its development progresses smoothly, we will apply for its manufacturing and sales approval in FY 2020, and in the following year we expect to obtain the manufacturing and sales approval.
If we confirm its efficacy on the other diseases in non-clinical trials, we will conduct clinical trials in humans for such diseases in order to expand its indications.
The Tonomachi CPC was established as a manufacturing site of the product for these purposes.
Our business is research, development and manufacturing of Muse cell product as the regenerative medicine.
We are expecting it as a manufacturing facility of the products for clinical trials, and also obtaining data of the product for submission of its manufacture and sales approval, and finally as the manufacturing site of the product for sales.
Research and business plans and implementation
What we will develop here to establish how to stably produce the product.
Since the cells are alive, we must consider: how to obtain its starting material, how to increase the cells, how to purify the cells, and how to store the cells.
We need to develop new methods which are not applied to the conventional medicines in the past.
The big difference compared with current medicines is sterilization, that is, conventional methods are not applicable to kill bacteria in the product.
Therefore, it is very important to consider how to keep its sterility and to prevent bacteria contamination in the course of its manufacturing.
Also, we handle the living cells, so we should pay close attention to the environment and safety as well. We would like to supply the product that is easy for everyone to use.
New discoveries and characteristics
Our Muse cells were discovered by Professor Dezawa of Tohoku University in 2010 and are the pluripotent stem cells present in our body.
The term “pluripotent stem cell” is a rare word, but it means that the cell has capability to become various cells of the body.
When Muse cells are injected intravenously, they receive a signal from a diseased site, accumulates at the site, and then Muse cells are spontaneously differentiated into a tissues of the site and repairing those.
As I mentioned earlier, practical-use is also expected. Because Muse cells exist in the body, so, they might be safe.
The product is manufactured from healthy donor’s cells, can be cryopreserved.
And its administration is done by an injection, we would expect it to be used by medical institutions, even, not advanced facility, for example, to conduct surgery.
For Muse cell product, we have confirmed its efficacy in pre-clinical studies, and we are currently evaluating its safety and efficacy in patients in the clinical trials.
Our Muse cells business has a potential to offer new treatment opportunities for diseases that cannot be cured by current treatment.
At the moment, we are conducting clinical trials in humans for three diseases: acute myocardial infarction, cerebral infarction, and epidermolysis bullosa.
And we are evaluating the product’s safety and efficacy.
Continuously, we will conduct clinical trials for further indications after confirming the product’s efficacy in various non-clinical studies.
For regenerative medicine, advanced facilities may be required. But, we believe that our products can be used without such facilities, and regenerative medicine are became more familiar therapy to contribute to the recovery of the body's functions.
Expectations for King Skyfront
King Skyfront has very big advantages because it is located very close to Haneda International Airport.
Firstly, the international airport is convenient for providing the product outside Japan. In this sense, it is a very advantageous place for international development.
In addition, we believe that it will be much easier to receive researchers/scientists from not only Japan but also overseas.
Furthermore, at King SkyFront, many regenerative medical companies are accumulated. Among these companies, exchanging know-how and knowledge will be expected and also new business opportunities will be found.