NEW YORK – Grace Science on Tuesday said it has gained permission from the US Food and Drug Administration to begin testing its gene therapy for NGLY1 deficiency in humans.
FDA clearance of Grace Science's investigational new drug application allows the Menlo Park, California-based biotech firm to initiate a Phase I/II/III trial for GS-100. The gene replacement therapy encodes a full-length version of the NGLY1 gene using an adeno-associated virus serotype 9 vector and is administered by intracerebroventricular infusion.
In the open-label, single-arm, dose-finding study, Grace Science will assess GS-100's long-term safety and efficacy in patients between 2 years and 18 years old.
Patients with NGLY1 deficiency suffer from debilitating neurological symptoms including global developmental delay, cognitive impairment, and movement disorders. "This is an important milestone for NGLY1 deficiency families, as well as for our company," Grace Science Cofounder Carolyn Bertozzi said in a statement. "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.”
The FDA and the European Medicines Agency previously granted GS-100 orphan drug designation. The gene therapy also has rare pediatric disease designation from the FDA.