CDG Care Awards $60,000 in Research Grants for PIGA-CDG Gene Therapy Development

Featured on CDG Care’s website:

CDG Care has awarded approximately $60,000 to the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital in Columbus, Ohio and Osaka University in Japan for the development of a gene therapy program for the treatment of PIGA-CDG, also known as Multiple Congenital Anomalies-Hypotonia-Seizures Syndrome Type 2 (MCAHS2).

This grant builds upon the successful collaboration established in 2018 between PIGA-CDG families and Osaka University. Researchers from AWRI at Nationwide Children’s will develop and manufacture gene therapy vectors for testing in pre-clinical studies with the goal of developing a gene therapy to help treat PIGA-CDG.

The gene therapy vectors to be manufactured by Nationwide Children’s will be sent to Osaka University for testing in PIGA cell lines and mouse models to evaluate efficacy in treating PIGA-CDG. With the additional funding from this research grant, researchers from Osaka University will also generate and maintain PIGA knock-in mice that can be used for in-vivo testing of the PIGA-gene therapy vectors as well as other compounds being evaluated for treatment of PIGA-CDG.

To learn more about PIGA-CDG and the research and advancements being made in PIGA-CDG, please visit www.piga-cdg.com.