RARE Daily

FDA Clears Grace’s IND to Initiate a Phase 1/2/3 Trial of Gene Therapy for the Treatment of NGLY1 Deficiency

November 7, 2023

Rare Daily Staff

Grace Science said that it has received FDA clearance of its Investigational New Drug application for GS-100, an AAV9 gene replacement therapy for the treatment of NGLY1 deficiency.

NGLY1 deficiency is a serious, life-threatening disease with no approved therapy. Patients with this disease suffer from a lifetime of debilitating symptoms, including global developmental delay, cognitive impairment, (hypo)alacrima, movement disorders, and other neurological symptoms.

The clinical trial will utilize a single-protocol and an innovative phase 1/2/3 trial design that includes an open-label, single arm, dose-finding study to investigate the long-term safety and efficacy of GS-100 administered by intracerebroventricular infusion to NGLY1 Deficiency patients 2 to 18 years of age.

GS-100 is a recombinant, single-stranded AAV9 vector that encodes a full-length version of the human NGLY1 gene. GS-100 was previously granted Orphan Drug designation  by the FDA and by the European Medicine Agency (EMA) in 2021. GS-100 was also granted Rare Pediatric Disease Designation by the FDA in 2021, providing the potential for a Priority Review Voucher upon marketing approval.

“This is an important milestone for NGLY1 Deficiency families, as well as for our company,” said Carolyn Bertozzi, co-founder of Grace Science. “This is the first Grace Science program to receive FDA clearance to enter the clinic and the first gene therapy clinical trial for NGLY1 deficiency.”

Photo: Carolyn Bertozzi, co-founder of Grace Science

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