Gene Therapy Research Institution (GTRI) is a pioneering entity in the field of gene therapy, focusing on the development of treatments for central nervous system (CNS) diseases and monogenic disorders. Operating as an academic research hub in Japan, GTRI collaborates with numerous researchers across the country, thanks to the expertise of Dr. Muramatsu, a leading figure in AAV vector research. The institution's primary programs target novel treatments for Sporadic ALS and Parkinson's disease, aiming to bring groundbreaking therapies to clinical practice. GTRI serves a diverse clientele, including academic researchers, medical professionals, and patients suffering from severe neurodegenerative diseases. The company operates in the biotechnology and healthcare market, utilizing a business model that combines research funding, grants, and donations to support its initiatives. Revenue is generated through collaborative research projects, licensing of intellectual property, and potential commercialization of successful therapies.

Keywords: gene therapy, CNS diseases, monogenic disorders, AAV vector, Sporadic ALS, Parkinson's disease, academic research, biotechnology, healthcare, neurodegenerative.