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

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