FDA Grants RMAT Designation to Grace Science’s Gene Therapy for NGLY1 Deficiency

Rare Daily Staff

The U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Grace Science’s GS-100, an experimental gene therapy for NGLY1 Deficiency, an ultra-rare genetic disorder with no approved treatments.

NGLY1 Deficiency is caused by mutations that prevent production of functional NGLY1 protein, leading to neurological symptoms, movement disorders, and developmental delays that appear in early childhood. Fewer than 100 cases have been identified worldwide, and there are currently no approved medicines for the condition.

GS-100 is designed to replace the missing or defective NGLY1 enzyme using an adeno-associated viral vector. Early data from Grace Science’s ongoing phase 1/2/3 clinical trial suggest the therapy may improve motor and cognitive function in affected children, including gains in sitting, standing, and walking with assistance, as well as greater attention and engagement.

The RMAT program, created to accelerate development of regenerative therapies such as gene and cell treatments, offers benefits including intensive FDA guidance, eligibility for accelerated approval, and priority review if certain criteria are met.

GS-100 has previously received Orphan Drug, Rare Pediatric Disease, and Fast Track designations. The FDA also accepted the therapy into its START Pilot Program, which aims to enhance communication between regulators and developers working on rare disease therapies.

“The FDA’s RMAT designation underscores the encouraging clinical data observed after GS-100 treatment,” said Matt Wilsey, co-founder and CEO of Grace Science. “This designation provides a clear and potentially streamlined regulatory path forward.”