Phase 1/2/3 GS-100 Gene Therapy Trial Initiates with First Patient Dosed

Grace Science, LLC has initiated a clinical trial for a novel gene therapy, GS-100, which targets NGLY1 Deficiency, a rare and severe genetic disorder. The open-label study is structured in three phases, beginning with a Phase 1/2 segment that will explore the dosage and the safety and efficacy of administering GS-100 via a single intracerebroventricular infusion. This segment will focus on pediatric patients between the ages of 2 and 18.

The third phase of the trial will evaluate the primary outcomes at the 52-week mark post-treatment, specifically examining the changes from baseline levels of the NGLY1 Deficiency biomarker, GlcNAc-Asn, in cerebrospinal fluid, and the motor subdomain of the Bayley Scales of Infant and Toddler Development, Fourth Edition (BSID-4).

The CEO and Co-Founder of Grace Science, Matt Wilsey, expressed that dosing the first patient is a significant step forward for the company and the NGLY1 Deficiency community. The urgency is underscored by the loss of sixteen community members since the beginning of 2020. Carolyn Bertozzi, Co-Founder of Grace Science, emphasized the collaborative nature of the study, highlighting the partnership between the scientific community and the patient community, and the dedication of the team in bringing this therapy to fruition.

GS-100 is a recombinant, single-stranded AAV9 vector designed to carry the full-length human NGLY1 gene. It has received several designations, including Orphan Drug Designation from both the FDA and the European Medicine Agency, Rare Pediatric Disease Designation from the FDA, and Fast Track Designation from the FDA.

NGLY1 Deficiency is characterized by early-onset, severe symptoms such as developmental delays, cognitive impairment, and neurological issues. There are currently no approved treatments for this life-threatening condition.

Grace Science, founded in 2017 by Matt Wilsey and Dr. Carolyn Bertozzi, is a biotechnology firm dedicated to developing therapies based on the NGLY1 enzyme's role in proteostasis. The company's extensive understanding of the NGLY1 pathway is crucial to its mission to treat NGLY1 Deficiency and other prevalent diseases.