Dr. Takamitsu Yano,PhD

Aiming to help patients lead more active and social lives...
Duchenne muscular dystrophy, caused by a genetic mutation, is a rare intractable disease that results in the gradual loss of muscles throughout the body. Dr.Takamitsu Yano, who invented a new therapeutic molecule for this disease in the course of basic research, has launched a biotech venture company venture with two of his friends and is working on the development of a pharmaceutical product. The nucleic acid therapeutic molecule that induces a new frameshift has the potential to bring about effects beyond “life support,” which is the limitation of existing drugs. Confirmation of efficacy at the cellular level and patent applications have already been filed. In this project, we aim to confirm the efficacy of the therapeutic molecule in a mouse study and take a step forward in the development step toward pharmaceutical realization.