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Grace Science, LLC Announces First Patient Treated in Phase 1/2/3 Trial of GS-100, an AAV9 Gene Therapy for NGLY1 Deficiency

MENLO PARK, Calif.--(BUSINESS WIRE)--Grace Science, LLC announced today that the first patient was dosed in an open-label Phase 1/2/3 clinical trial of GS-100, an AAV9 gene replacement therapy for the ...
Business Wire

Grace Science Receives U.S. FDA Orphan Drug Designation for GS-100, an AAV9 Gene Therapy for NGLY1 Deficiency

MENLO PARK, Calif.--(BUSINESS WIRE)--Grace Science, LLC announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GS-100, an investigational AAV9 gene ...