MENLO PARK, Calif.--(BUSINESS WIRE)--Grace Science, LLC announced today that it has received FDA clearance of its Investigational New Drug (IND) application for GS-100, an AAV9 gene replacement therapy for the treatment of NGLY1 Deficiency.
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 (ICV) infusion to NGLY1 Deficiency patients 2-18 years of age.
“Dr. Bertozzi and I started Grace Science to save lives across the rare disease community — and beyond. We knew that the best way to do that was to approach science and drug development in a different way,” said Matt Wilsey, Co-Founder and Chief Executive Officer of Grace Science, LLC. “Today’s news is an exciting validation of that approach. We would not be here without the hundreds of people who developed the drug from patients, families, and our incredible partners.”
“We are thrilled to obtain FDA clearance to advance GS-100 into the clinic and are excited about the prospect of what this new treatment may mean for NGLY1 Deficiency patients and families,” said Dr. Carolyn Bertozzi, Co-Founder of Grace Science, LLC. “This is an important milestone for NGLY1 Deficiency families, as well as for our company. 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.”
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 (ODD) 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.
About 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.
About Grace Science
Grace Science, LLC is a biotechnology company founded in 2017 by Matt Wilsey and Dr. Carolyn Bertozzi to develop novel therapies based on the function of NGLY1, a key enzyme involved in proteostasis. The company’s deep knowledge of the NGLY1 pathway is key to its ability to identify new ways of treating NGLY1 Deficiency as well as more common diseases. For more information about Grace Science, LLC and NGLY1 Deficiency, please visit gracescience.com.