T-CiRA & RIKEN To Tackle Rare Disease With iPS Cells

CiRA of Kyoto University, Takeda and RIKEN are collaborating to discover a drug for a rare genetic disorder called NGLY1 deficiency.

AsianScientist (Apr. 7, 2017) – The Center for iPS Cell Research and Application at Kyoto University (CiRA), Takeda Pharmaceutical Company Limited (Takeda) and RIKEN have entered a partnership 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 Dr. Tadashi Suzuki of RIKEN, who first identified the NGLY1 gene.

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, induced pluripotent (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 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 Yamanaka, who received a Nobel Prize in 2012 for his work on iPS cells.


Source: Takeda; Photo: Shutterstock.
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