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Font--more Large-- Japanese Tel: +81-3-3416-0181 ／ Reservation（Hospital）: 03-5494-7300 〈Monday～Friday（except national holiday）9A.M.〜5P.M.〉 Access Contact Us Sitemap Japan Council for Quality Health Care Site Search Patients & Families Hospital Introduction International Patients Medical partners Hospital Divisions Researchers Research Institute About Research Divisions Center for Clinical Research and Development About Departments Achievement Donation サイドメニュー--> Recruiting and Training サイドメニュー--> About NCCHD Message from the President Founding Principle / Our Mission History Facts & Figures Activity Patients & Families Hospital Introduction International Patients Medical partners Medical partners Hospital Divisions Researchers Research Institute Center for Clinical Research and Development Donation Donation Recruiting and Training Recruiting and Training About NCCHD Message from the President Founding Principle / Our Mission History Facts & Figures Activity Facilities Hospital Research Institute Clinical Research Access Contact Us Sitemap Japan Council for Quality Health Care Japanese トップ > English > About > Activity > gcp Gene & Cell Therapy Promotion Center Overview Leadership and Projects Consultations for industry and academia Japanese Overview Gene and cell therapy is an effective therapeutic option for childhood intractable diseases, such as inborn immunity and metabolism errors, neurological diseases, and blood diseases. Immune therapy as represented by chimeric antigen receptor T-cell (CAR-T) therapy is also used to treat childhood hematological malignancy. However, the therapies are still under development and need a new type of system to evaluate the safety and efficacy of implementing the therapies in a clinical setting. In addition, in compliance with the Cartagena Act, when using viral vectors, a special preparation is required for virus shedding in medical facilities. Therefore, the Gene & Cell Therapy Promotion Center was established on April 7, 2019, to support pharmaceutical companies and academia to properly administer gene and cell therapy in Japan through consultations on clinical trials. We hope our activities will help facilitate gene and cell therapy in Japan. Ongoing Gene and Cell Therapy Protocols at NCCHD Condition / disease Product Category Patients Leukemia KYMRIAH（R） Approved product 12 Hemophilia B PF-06838435 Clinical Trial 1 Spinal muscular atrophy ZOLGENSMA（R） Approved product 2 Duchenne Muscular Dystrophy SRP-9001 Clinical Trial 2 NCCHD Pamphlet Message from the President Founding Principle / Our Mission History Activity Facts & Figures About NCCHD NCCHD Pamphlet Message from the President Founding Principle / Our Mission History Activity Facts & Figures Return to top of page SitemapPrivacy Policy 2-10-1 Okura,Setagaya-ku,Tokyo 〒157-8535　Tel：+81-3-3416-0181FAX：03-3416-2222 © National Center for Child Health and Development All rights reserved.