Gene Therapy Research Institution Co., Ltd.
Development of gene therapy
Using adeno-associated virus (AAV) as a vector to carry therapeutic genes, we are developing gene therapy for intractable diseases for which no effective treatment has been available so far. We have already started clinical trials for AADC deficiency, Parkinson's disease, and amyotrophic lateral sclerosis (ALS).
Regenerative Medicine
- Drug Discovery/Drug Discovery Support