The first study of the “Rare Disease Research and Development Program based on patient-centered principle” that connects researchers with rare diseases and intractable disease patients has been completed.

KOTAI Biotechnology, Inc. (Kazuo Yamashita, CEO, hereinafter referred to as “KOTAI”) and 3H Holdings Inc. (Sho Ando, CEO, formerly CROee, hereinafter referred to as “3H”) are pleased to announce that the recruitment of research cooperation among autoimmune disease patients to “Study on specific immune conditions of autoimmune diseases”, which reached a capacity of 30 people on August 7, 2019, about one month from being started on July 1, 2019.

This is the first study on patient-centered R&D program for rare diseases (Patient Centricity in Rare Disease R&D Program, hereinafter referred to as “PCRD²”) jointly conducted by the University of Tokyo and 3H Holdings Inc., the purpose of which is to promote research and development by connecting patients with intractable diseases who are seeking for new therapies to researchers who do not have access to the testing samples necessary for their research.

This result is an example that patients who have unmet medical needs are eager to collaborate on research and development of new therapies, while patient / citizen participation and patient centricity are attracting attention. Since then there have been many requests for cooperation, we are currently expanding the recruitment capacity and extending the period, and at the same time conducting a questionnaire survey at the University of Tokyo in order to promote better research and development of rare diseases based on patient-centricity. We have also started pre-registration for research cooperation.

• Study on specific immune conditions of autoimmune diseases：https://raresnet.com/special/pcrd2-pro01/
• Awareness survey and pre-registration：https://raresnet.com/special/pcrd2/

The comments of each parties concerned about this study and the results are as follows.

Voices of patients who cooperated in the study

“I participated in the study because I wanted to know more about the mechanism related to my illness, and by investigating it, I understood the relationship with other illnesses and hope I can be of service. I sincerely hope that the day will come when better drug for my illness will be developed soon and everyone can become “drug-free”. “

Kyoko Imamura, Project Professor, Social Cooperation Program of IT Healthcare, Graduate School of Pharmaceutical Sciences, The University of Tokyo

“In the Study of Specific Immune Conditions of Autoimmune Diseases” by KOTAI which is the first project of PCRD² by 3H Holdings Inc. and our laboratory, we were provided samples and information from patients at three times of the speed than we expected. It was a fundamental study of the immune repertoire technology that is difficult even for the researchers, but I felt a strong desire of the patients to jointly develop with the researchers as well as the expectations for new drugs.”

Kazuo Yamashita, CEO, KOTAI Biotechnologies, Inc.

“I am very pleased that our clinical research, the first project of PCRD², has successfully completed the recruitment much sooner than we expected due the support of many patients. We strongly recognize the difficulty and importance of the challenges of healing rare diseases and the usefulness of PCRD² as one of the solutions. In order to develop the research and the framework of PCRD² further, we will provide feedback to the patients on the results of our research and show them the path to development of diagnostics and treatments.”

Daisuke Maki, 3H Life Science Research Institute

“I strongly felt that both patients and researchers have the same desires to have a new treatment as soon as possible. It is unfortunate for everyone that there is no access to the opportunity to cooperate each other. We are very pleased that the 3H Group has contributed to the realization of Patient Centricity in the unmet medical field. We will continue to develop and implement programs appropriately to solve such problems. “

About research

※The following is an update of the news release delivered on July 1, 2019.

Outline of research

Immune cells, T cells and B cells as target this time, are deeply involved in the onset and the progress of diseases in autoimmune diseases, while at the same time playing an important role in many diseases, such as cancer and infectious diseases. We will study the following two issues by analyzing the function of immune cell receptors called T-cell receptors and B-cell receptors), which play an important role as limbs of disease-related T cells and B cells:

1. We will clarify the causes of “rare intractable autoimmune diseases” where the causes of the diseases are not clear and promote the creation of new therapeutic agents.
2. We will explore the use of effective therapeutic agents for other diseases with regard to “rare intractable autoimmune diseases” that do not have therapeutic agents.

Target diseases

Those who are suffering from the disease and are currently undergoing treatment
Target diseases: Bechet’s disease, Ankylosing Spondylitis, Psoriasis, Polymyositis, Dermatomyositis, Adult Still’s Disease, Rapidly Progressive Glomerulonephritis, Ulcerative colitis, Crohn’s disease, Rheumatoid Arthritis

As of August 9, 2019, additional plans will be made in the future
※For more information, please check the reception page.

Research results that can be provided to patients

After the analysis is completed, we will make a briefing the patients, who wish to have information, about the rough profiles of similarities and differences of each patient’s immunity in the study as a summary of the results obtained from the trial.

This joint research is the first step in finding a therapeutic agent together. Although this joint research period will be short and preliminary, we would like to conduct a long-term joint research not only to find new drugs to treat diseases in the long term, but also to determine what kind of lifestyles and existing medications can stabilize your conditions.

The contents of “Study of Specific Immune Conditions of Autoimmune Diseases” in the information website “RateS.” for Intractable disease and rare disease

Content of research and application：https://raresnet.com/special/pcrd2-pro01/

About PCRD² (https://3h-holdings.co.jp/20190517/)

This program supports the participation of patients to research and development in the area of rare diseases and intractable diseases and the promotion of research. Patients will provide testing samples and researchers will bear the costs necessary for providing information to patients within the range that does not affect intellectual property and the expenses for using the samples.

About Social Cooperation Program of IT Healthcare, Graduate School of Pharmaceutical Sciences, The University of Tokyo(https://plaza.umin.ac.jp/ithc-utokyo/)

The course is conducted by six joint research companies, including the Graduate School of Pharmaceutical Sciences, The University of Tokyo and 3H Holdings Inc. to conduct research on drug development and innovation of proper use through ICT utilization.

• Construction of regulatory science of data reliability necessary for conducting home and remote clinical trials
• Programming of ICT education required by pharmacists, systematization of medication information, and establishment of a system to support pharmacies
• Programing of education on drug development and data utilization for leadership development to promote patient group activities
• Realization of disease epidemiology and medical innovation by exploratory analysis of Real World Data

Organization Overview

KOTAI Biotechnology, Inc.

Location: 2F, Bldg. B of Office for Industry-University Co-Creation,
Osaka University, 2-1 Yamadaoka, Suita, Osaka
Representative: Kazuo Yamashita, CEO
Website: https://www.kotai-bio.com/

3H Holdings Inc.

Location of headquarters: Ikebukuro YS building 2F, 1-13-23 Minami-Ikebukuro, Toshima-ku, Tokyo
Presentative: Sho Ando, CEO
Nature of business: Group Business Administration, Research on Life Science