Agilis GTRI Japan Inc.

AADC deficiency is an autosomal recessive inherited disease where aromatic L-amino acid decarboxylase (AADC), an enzyme responsible for synthesizing the neurotransmitters, does not work due to congenital gene mutation. It causes symptoms such as insufficient autonomic function and difficulty in moving the body. It is an intractable disease and there are only around 100 reported cases in the world. Katsuhito Asai, President of AGJ, says, “There are only six known patients ブラック ジャック トランプ やり方apan so far.” In April 2016, the disease was included ブラック ジャック トランプ やり方apan’s list of intractable diseases. In contrast, there are 150,000 Parkinson’s disease patients ブラック ジャック トランプ やり方apan. If AGJ's gene therapy drugs are approved, it will open the way to treatment for intractable diseases for which no cure exists such as ADDC deficiency and Parkinson’s disease.

The parent company, Agilis Biotherapeutics, LLC (hereinafter called "Agilis"), is a bio-venture company established in Cambridge, Massachusetts, the US in 2013. It is a world leader in the clinical development of ブラック ジャック トランプ やり方y for AADC deficiency and has provided treatment to the largest number of patients in the world. Agilis is recognized as having the most extensive experience and advanced technologies in AADC ブラック ジャック トランプ やり方y. In December 2015, the ブラック ジャック トランプ やり方y Research Institution Co., Ltd., a Japanese bio-venture company conducting research in the same field (hereinafter called “GTRI”), offered a partnership to Agilis.

Possessing top gene therapy technology using AAV vectors, GTRI is one of only a few companies in the world developing gene therapies in the central nervous system and neurology fields. The decisive factor for Agilis in accepting the partnership with GTRI was “the pipelines(*2) that GTRI has ブラック ジャック トランプ やり方apan and the progress of their research and development of the manufacturing technology of such pipelines,” said Mr. Asai. AGJ aims to strengthen the foundation of gene therapy drug development in Asia utilizing the advanced manufacturing technologies of Japanese companies and the experience of Agilis in gene therapy. ブラック ジャック トランプ やり方uly 2016, about a half year after the offer of partnership, a business cooperation agreement was concluded through consultation provided by JETRO, and a joint venture, AGJ, was established in August of the same year.

Currently, 10 staff members, including Mr. Asai and eight researchers, are preparing for the manufacture of gene therapy drugs in the Life Innovation Center. Their first goal is to obtain approval of compliance with GCTP, a standard for manufacturing and quality management of regenerative medicine products. Mr. Asai said, “We are making every effort to prepare for obtaining approval of GCTP compliance. Without this approval, we cannot start clinical trials. As this process takes some time, we are also concurrently studying how to expand the laboratory's current manufacturing scale to a commercial production level.” Upon obtaining approval, the company will then prepare for international multicenter clinical trials in the center. Mr. Asai talked about his passion for manufacturing gene therapy drugs, saying, “Using the international multicenter clinical trial framework, we can utilize the results of trials ブラック ジャック トランプ やり方apan in our application for approval in the US and Europe. The standards of most other countries are no more stringent than those of Japan. We want to make this center a regional base for Asia and Europe as well as a base of manufacturing and marketing in the future.” The company has received inquiries about the clinical trial ブラック ジャック トランプ やり方apan from overseas patients eager to be considered as candidates.”

Not only is this cutting-edge treatment being developed by AGJ expected to be the sole highly-safe treatment for AADC deficiency and Parkinson’s disease, but the application of its technology is also expected to establish treatment strategies for other genetic diseases.

(Interviewed ブラック ジャック トランプ やり方une 2017)