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Vol.16,September 2019

Katsuhito Asai

Gene Therapy Research Institution Co.,Ltd CEO

Gene therapy

Gene therapy is a technology that puts genes directly into human cells and cures the disease. It was developed in the United States in the middle of the 90’s, and many technological improvements since then.

Now the number of applications for gene therapy is increasing compared to cell therapy and regenerative medicine in the United States. It is becoming a boom.

In terms of technological development and innovation, the first is the use of the so called Adeno-Associated Virus (AAV) at the time of gene transfer. Gene transfer using this AAV vector is very efficient and safe. So far, clinical research or clinical trials have been conducted in over 200 people worldwide, and there has been no accident using this AAV vector.

In the first place, AAV vectors are non-pathogenic viruses, and when performing gene transfer, they use only the function of introducing the target gene sequence into the target cells. It is not pathogenic originally, but all unnecessary sequences are removed. So the possibility of side effects is extremely low.

So what is the benefit of this gene therapy compared to the development of ordinary drugs for patients. First, it is important that drugs could not be directly delivered to neurons in the brain or into the brain. This was not possible before. Now, you can directly introduce the underlying gene sequence into cells in the brain.

As a result, this method can cure severe atrophic diseases such as amyotrophic lateral sclerosis (ALS) that have never been treated before, or enable radical treatment of Parkinson’s disease, and also Alzheimer’s. In the future, we will be able to treat Alzheimer’s disease, a major social problem, with our technology, including prevention. In that respect, I think there are benefits for patients.

Another important thing is that once drugs are administered, the effects last for a lifetime. In other words, once administered, the disease will improve without the need for subsequent medication. If you also point out that there are no side effects, it may be a dreamlike treatment for patients.

Characteristics of the Gene Therapy Research Institution

The original research of our company is based on research conducted by Professor Muramatsu of Jichi Medical University that started in the 1990s. Therefore, we are collaborating with many Japanese universities including Jichi Medical University, and I think that our company is a hub for research on gene therapy using AAV in Japan. In the United States, there are many companies that do AAV gene therapy, and more than 20 companies are listed on the stock market, but when compared with such companies, the number of programs being developed is very large.

Regarding Muramatsu’s invention, we have our own AAV vector, that is, our own technology to introduce genes. Our technology is much more efficient than gene transfer methods developed in the United States, and this is our strength. In addition, we are currently installing and operating manufacturing facilities in Kawasaki City that meet the manufacturing standards for pharmaceutical products. We are the first facility in Japan to make a formulation for gene therapy of this AAV vector, and have obtained manufacturing permission.

Status of current research

Our leading development program will be for gene therapy for sporadic ALS, and we are planning to conduct a clinical trials next year.

Next, will follow trials for Parkinson’s disease in the second half of next year. We plan to launch trials in Japan, the US, and Europe simultaneously.


First of all, we want to prove to our patients that our treatment method is effective and safe for diseases for which there has never been a fundamental treatment. On that basis, I would like as many people as possible to undergo this gene therapy. In fact, there are not many companies that have commercialized AAV in Japan.

Next month, it is expected that the European pharmaceutical company Novartis will be the first in Japan to receive approval for AAV-based gene therapy. Following that, we will aim to obtain approval for the intractable diseases of the nervous system as I explained. Unfortunately, pharmaceutical companies in Europe and the US have set the fee for a single treatment to be more than 200 million yen. At the stage of our approval, we are continuing to develop technology to enable a cost of 1/10 to 1/00 of this 200 million yen.

Expectations for Kingskyfront

Kingskyfront is an environment where you can focus on research, development, and manufacturing. We are currently collaborating with other companies in the Kingskyfront facilities. It is good for us to be able to do research and develop in this hub where there is a high concentration of bio-industries.

Now we have only one experimental facility and one manufacturing facility, but we have to make as many gene therapy preparations as possible for various diseases, so we are expanding factory for manufacturing. We plan to build the second and third plants in the Kingskyfront.