Grace Science, LLC has initiated a Phase 1/2/3 clinical trial for GS-100, a gene therapy aimed at treating NGLY1 Deficiency.

The trial involves dose-escalation and will evaluate the safety and effectiveness of a single ICV infusion in a pediatric population aged 2-18 years.

Efficacy will be measured by changes in biomarkers of disease activity and motor development at a one-year mark post-treatment.

GS-100 has been granted Orphan Drug and Fast Track Designations, indicating its potential to address an unmet medical need.

NGLY1 Deficiency is a serious condition without any approved treatments, and GS-100 presents a hopeful option for those affected.