Grace Science Initiates Phase 1/2/3 Trial with First Patient Treated for NGLY1 Deficiency Using GS-100 AAV9 Gene Therapy

Grace Science, LLC has initiated a clinical trial for GS-100, a gene therapy developed to treat NGLY1 Deficiency, with the first patient receiving the treatment. The open-label study is divided into two parts: the initial phase focuses on dose escalation and evaluating the safety and efficacy of GS-100 through a single intracerebroventricular infusion for patients aged between 2 and 18 years. The third phase will measure the primary outcomes at 52 weeks post-treatment, including changes in the NGLY1 Deficiency biomarker and motor development as assessed by the Bayley Scales of Infant and Toddler Development, Fourth Edition.
More information on the clinical trial, registered as NCT06199531, can be accessed on ClinicalTrials.gov. Matt Wilsey, CEO of Grace Science, emphasized the significance of this milestone for the company and the NGLY1 Deficiency community, highlighting the urgency given the loss of sixteen community members since the start of 2020. Carolyn Bertozzi, Co-Founder, expressed gratitude to the patient community for their role in advancing the therapy.
GS-100 is an AAV9 vector designed with the complete human NGLY1 gene and has received various designations, including Orphan Drug status from the FDA and EMA, Rare Pediatric Disease status, and Fast Track status. NGLY1 Deficiency is a severe, life-threatening condition characterized by early-onset symptoms such as developmental delays and cognitive impairments. Grace Science, established in 2017 by Wilsey and Bertozzi, specializes in therapies targeting the NGLY1 enzyme, which plays a crucial role in cellular protein management. The company's expertise in the NGLY1 pathway aids in developing treatments for both NGLY1 Deficiency and other prevalent diseases. 

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