Most hereditary genetic diseases are rare diseases, which belong to the “long tail” of diseases with small numbers of patients. However, even though the number of patients with each individual disease is small, there are 400 million patients worldwide suffering from rare diseases as a whole. The development of treatments for these rare diseases has been avoided in the past because the development costs and development periods are enormous, and 95% of rare diseases still have no treatment.

We believe that new technologies such as gene therapy and genome editing are a breakthrough that will enable the development of effective treatments for hereditary genetic diseases. The first reason is that the same methodology can be reproduced across different diseases. The mechanisms that cause genetic diseases are often similar from disease to disease, and this provides many opportunities to apply the same approach to other diseases. The second reason is that the mechanisms that govern genes are highly conserved between humans and animals, so it is possible to predict the effects of clinical trials with a high degree of accuracy based on animal testing.

We have developed a new genome editing technology called CRISPR, and have created a new platform technology called CRISPR-GNDM® that can be used to efficiently and effectively create treatments for genetic diseases. Our vision is that this technology will lead to the development of new treatments for genetic diseases one by one.

EVERY LIFE DESERVES ATTENTION

We believe that no disease should be neglected just because there are not many patients. We will use our technology to solve the problem.

Haru Morita
President and CEO