Grace Science, LLC Selected by FDA to Participate in the START Pilot Program for GS-100 Gene Therapy for NGLY1 Deficiency and Announcement of the Successful Treatment of the 2nd Patient

June 3, 2024 |

3 min read

Grace Science, LLC announced today that the IND for GS-100, an AAV9 gene therapy for NGLY1 Deficiency, was accepted into the FDA’s Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program.

MENLO PARK, Calif.--(BUSINESS WIRE)-- Grace Science, LLC announced today that the IND for GS-100, an AAV9 gene therapy for NGLY1 Deficiency, was accepted into the FDA’s Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program.

The purpose of the START Pilot Program is to provide enhanced communications with the FDA to further accelerate the pace of drug development for rare diseases and to provide a mechanism for addressing clinical development issues. These enhanced communications will include an initial meeting and subsequent ad hoc communications with the FDA.

More information about the START Pilot Program can be found on the FDA website at https://www.fda.gov/news-events/press-announcements/fda-launches-pilot-program-help-further-accelerate-development-rare-disease-therapies.

Grace Science also announced that the 2nd NGLY1 Deficiency patient was successfully treated with GS-100 in May. Both the 1st and 2nd patients (first cohort) treated with GS-100 continue to do well with no drug-related adverse events to date. A dose escalation is planned for the 3rd patient (second cohort) later this summer provided all safety signals remain positive in the first cohort.

Matt Wilsey, CEO & Co-Founder of Grace Science, LLC, stated that “Grace Science is appreciative of the opportunity to be one of the first companies to be accepted into the FDA’s START pilot program.” Mr. Wilsey went on to say, “We look forward to frequent conversations with the FDA about how best to streamline and accelerate the development of GS-100 for NGLY1 Deficiency.”

Dr. Carolyn Bertozzi, Co-Founder of Grace Science, LLC, added, “We believe GS-100 has the potential to improve the way drugs are discovered and developed. The START program will help us not only improve the lives of patients suffering from NGLY1 Deficiency, but also blaze a trail for other diseases that are both rare and common.”

About the GS-100 Clinical Trial

This is a Phase 1/2/3 open-label, single arm, dose-finding study to investigate the long-term safety, tolerability, and efficacy of GS-100, an AAV9 gene replacement therapy for NGLY1 Deficiency.

The Phase 1/2 part of the study will investigate dose-escalation and the safety and efficacy of a single intracerebroventricular (ICV) infusion in patients aged 2-18 years at up to three dose levels.

The Phase 3 part of the study will assess the following co-primary outcomes at 52 weeks following treatment at the selected dose: 1) change from baseline of the NGLY1 Deficiency Primary Disease Activity Biomarker, GlcNAc-Asn (GNA, or N-acetylglucosamine) in cerebrospinal fluid, and 2) change from baseline in the motor subdomain of the Bayley Scales of Infant and Toddler Development, Fourth Edition (BSID-4).

Further details regarding the Phase 1/2/3 study can be found at https://clinicaltrials.gov/study/NCT06199531

About NGLY1 Deficiency

NGLY1 Deficiency is a serious, life-threatening disease with no approved therapy. Patients with this disease suffer from debilitating symptoms that present early in life, 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.