U.S. FDA Grants RMAT Designation to GS-100, Grace Science’s Gene Therapy to Treat NGLY1 Deficiency

MENLO PARK, Calif.--(BUSINESS WIRE)--Grace Science, LLC, a biotechnology company founded to develop novel therapies based on the function of NGLY1, announced today that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to GS-100 for the treatment of NGLY1 Deficiency. GS-100 is an investigational AAV9 gene replacement therapy in clinical development to treat NGLY1 Deficiency, an ultra-rare genetic disease with no approved therapy.

“GS-100 is the first and only treatment option in development specifically for NGLY1 Deficiency,” said Carolyn Bertozzi, Ph.D., co-founder and Scientific Advisory Board member of Grace Science.

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The RMAT designation for GS-100 is based on early clinical evidence from an ongoing open-label, single arm, dose-finding Phase 1/2/3 clinical trial, which is evaluating the long-term safety, tolerability and efficacy of GS-100 in patients with NGLY1 Deficiency. To date, improvements in motor function and cognitive skills have been detected in subjects treated with GS-100 for at least 52 weeks. These include gains related to the ability to sit, stand, or walk with assistance and improvements in caregiver interaction and attention following GS-100 treatment.

“The FDA’s RMAT designation emphasizes the encouraging clinical data after GS-100 treatment,” said Matt Wilsey, CEO and co-founder of Grace Science. “This designation is important because it provides a clear and potentially streamlined regulatory path forward. We look forward to working with the FDA to accelerate the clinical development of GS-100.”

“GS-100 is the first and only treatment option in development specifically for NGLY1 Deficiency,” said Carolyn Bertozzi, Ph.D., co-founder and Scientific Advisory Board member of Grace Science. “The RMAT designation marks an important milestone, and we are grateful to the families, clinicians, researchers and Grace Science team members who have helped us reach this point in our journey to bring GS-100 to children with this life-threatening disease who urgently need a treatment.”

The RMAT designation program is designed to expedite the development and review of regenerative medicine therapies, including gene therapies, that demonstrate the potential to address serious, life-threatening diseases based on preliminary clinical evidence. The designation provides important benefits in the drug development process and is designed to facilitate and expedite development and regulatory review, including accelerated approval, if relevant criteria are satisfied.

About GS-100

GS-100 is an investigational adeno-associated viral vector serotype 9 (AAV9) gene replacement therapy in clinical development to treat children with NGLY1 Deficiency. The viral vector encodes a full-length, functional version of the human NGLY1 protein.

The FDA previously granted Orphan Drug Designation to GS-100 in 2021, Rare Pediatric Disease Designation in 2021, and Fast Track designation in 2024. The FDA also accepted the Investigational New Drug (IND) application for GS-100 into its START (Support for clinical Trials Advancing Rare disease Therapeutics) Pilot Program in 2024, which is intended to provide enhanced communications with the FDA to further accelerate the pace of drug development for rare diseases.

About the GS-100 Phase 1/2/3 Clinical Trial

The open-label, single arm, dose-finding Phase 1/2/3 clinical trial is investigating the long-term safety, tolerability, and efficacy of GS-100 in 10 patients with NGLY1 Deficiency. The Phase 1/2 dose escalation part of the study investigated 4 different dose levels. The Phase 3 part of the study is assessing the safety and efficacy of subjects treated at the 1e15 vg/individual dose of GS-100. Additional information about the trial (NCT06199531) can be found at www.clinicaltrials.gov.

About NGLY1 Deficiency

NGLY1 Deficiency is an ultra-rare genetic disease caused by loss of function mutations in the NGLY1 gene. This serious, life-threatening disease primarily affects children and manifests as a complex neurological syndrome. Those affected experience debilitating symptoms that present early in life, including global developmental delay, cognitive impairment, abnormal tear production (alacrima), movement disorders, and other neurological symptoms. There is no FDA-approved therapy for NGLY1 Deficiency.

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.