The Center for iPS Cell Research and Application at Kyoto University (CiRA), Takeda Pharmaceutical Company Limited (Takeda) and RIKEN announced today that they will work together to search for a drug for NGLY1 deficiency. NGLY1 is a gene that encodes N-glycanase, an enzyme that catalyzes the deglycosylation of glycoproteins.

This project is a part of the Takeda-CiRA Joint Program for iPS Cell Applications (T-CiRA), which CiRA and Takeda announced in April 2015, and will be spearheaded by Tadashi Suzuki of RIKEN, who first identified the NGLY1 gene, as a principle investigator.

NGLY1 deficiency is a genetic rare disorder caused by mutations in the NGLY1 gene, and is characterized by a variety of symptoms including developmental and motor disorders, seizure and decreased production of tears. This project was selected based on the policy of T-CiRA to deliver innovative therapeutic options to solve high unmet medical needs. The project will combine basic research at RIKEN, iPS cell technology developed by Professor Shinya Yamanaka's group at CiRA, and Takeda's platforms for drug discovery to develop a treatment for NGLY1 deficiency for which no remedy has been established yet.

Team Leader Tadashi Suzuki, who is responsible for this project, said that finding a cure for NGLY1 deficiency is his mission. "It is a privilege to be able to apply our basic research to drug discovery. I will do my best to bring a therapy to patients as soon as possible thanks to the cooperation from Takeda and CiRA and will enhance our established collaboration with the Grace Science Foundation to find a cure for NGLY1 deficiency."

"Team Leader Suzuki is conducting research on the NGLY1 gene, which is important for maintaining the proper functioning of proteins in a cell. I hope that the knowledge he has accumulated and the T-CiRA partnership will rapidly bring us closer to a cure for NGLY1 deficiency," said CiRA Director Shinya Yamanaka, M.D., Ph.D., who received a Nobel Prize in 2012 for his work on iPS cells.

"I am pleased to hear that Dr. Suzuki is participating in the T-CiRA program. He is the world's leading researcher on NGLY1. Collaboration between RIKEN, a globally renowned research institution, and T-CiRA will produce much progress in research," said Seigo Izumo, Global Head of the Regenerative Medicine Unit of Takeda and Takeda's Chief Advisor to Professor Yamanaka.

• Information about this project
• T-CiRA website