Grace Science Initiates First Dose Administration of GS-100 Gene Therapy

Grace Science administered the initial dose of their gene therapy prospect to the first participant in an open-label early-stage trial. GS-100 is an AAV9 gene therapy designed to address NGLY1 deficiency.  

NGLY1 deficiency is a rare genetic disorder characterized by the impaired function of the N-glycanase 1 enzyme. Individuals with NGLY1 deficiency experience a range of symptoms, including developmental delays, movement abnormalities, and a lack of tears.  

The candidate has obtained orphan drug designation from the FDA in the United States and the European Medicines Agency.  

The initial research studies will analyse increased dosages and evaluate the candidate risks and efficiency.  

Carolyn Bertozzi, the cofounder of Grace Science, stated, “This study is a true partnership between the scientific community and the NGLY1 patient community.”  

Bertozzi went on to add, “The team has worked incredibly hard to reach the goal of treating the first NGLY1 deficiency patient with this potentially life-changing therapy.”  

Matt Wilsey, the head executive at Grace Science, stated, “We believe this drug will improve the lives of individuals living with this devastating disease. Time is precious as our community has lost 16 individuals since January 2020.”  

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