Gene Therapy Research Institution

Building
a new future of
gene therapy

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GTRI is building a world-leading gene therapy company addressing a broad range of CNS and monogenic disorders.

Gene therapies are currently being introduced into clinical practice and I am sure that they have promising medical and economic benefits. I have seen many patients who suffer from serious neurodegenerative diseases. Even as I speak, a number of patients are awaiting radical gene therapy treatment. Accordingly, I would like to implement the therapy in a practical clinical setting as soon as possible. By making the technology available, I shall improve their QOL and prolong their comfort and happiness. Gene therapy is effective means for achieving these goals and I would strongly like to put it into practice immediately, or in the near future, if possible.

PIPELINE

Pipeline

Diverse portfolio of internal programs for CNS diseases and monogenic disorders as academic research hub in Japan. Because Dr. Muramatsu is one of the few researchers of AAV-vector in Japan, many researchers who have research seeds from all over Japan offer him joint research. Lead internal programs are novel treatments for Sporadic ALS and Parkinson's disease.

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