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. T
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