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Gene & Cell Therapy Promotion Center


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 KYMRIAHR Approved product 7
Hemophilia B PF-06838435 Clinical Trial 1
Spinal muscular atrophy ZOLGENSMAR Approved product 1

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