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Grace Science Receives FDA Approval for IND Application to Commence Phase 1/2/3 Trial of GS-100, an AAV9 NGLY1 Gene Therapy, for NGLY1 Deficiency Treatment

Wednesday, November 08, 2023

Grace Science, LLC has announced that the FDA has granted clearance for its Investigational New Drug (IND) application for GS-100, an AAV9 gene replacement therapy designed to treat NGLY1 Deficiency.

The clinical trial will employ an innovative Phase 1/2/3 trial design, featuring a single-protocol and an open-label, single-arm, dose-finding study. This study is intended to evaluate the long-term safety and effectiveness of GS-100 when administered via intracerebroventricular (ICV) infusion to patients aged 2-18 with NGLY1 Deficiency.

Grace Science, LLC, expressed their commitment to saving lives within the rare disease community and their unique approach to science and drug development. He views the FDA's clearance as significant validation of their approach and acknowledges the collaborative effort that has gone into developing the drug, involving patients, families, and partners.

Grace Science, LLC, shared her excitement about advancing GS-100 into clinical trials and the potential benefits it may bring to NGLY1 Deficiency patients and their families. She emphasized that this milestone is not only a crucial step for NGLY1 Deficiency patients but also a significant achievement for their company. It marks the first Grace Science program to gain FDA clearance for clinical trials and the first gene therapy clinical trial designed for NGLY1 Deficiency.

GS-100 is described as a recombinant, single-stranded AAV9 vector that contains a full-length human NGLY1 gene. It has previously received orphan drug designation (ODD) from both the FDA and the European Medicine Agency (EMA) in 2021. Additionally, in 2021, the FDA granted it Rare Pediatric Disease Designation, which offers the potential for a Priority Review Voucher upon marketing approval.

NGLY1 Deficiency is a severe, life-threatening condition currently lacking an approved therapy. Patients with this disease experience a range of debilitating symptoms throughout their lives, including global developmental delay, cognitive impairment, (hypo)alacrima, movement disorders, and various neurological symptoms.



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