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

‘It Will Consume Your Life’: 4 Families Take On Rare Diseases

This Father Founded A Medical Research Startup To Save His Kid’s Life

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

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

Grace Science Announces FDA Clearance of Investigational New Drug (IND) Application to Initiate a Phase 1/2/3 Trial for the Treatment of NGLY1 Deficiency with GS-100, an AAV9 NGLY1 Gene Therapy

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

Ngly1 rats develop neurodegenerative phenotypes and pathological abnormalities

Characterized and demonstrated the power of the NGLY1 Deficiency biomarker, GNA.

The calculated U.S. incidence of NGLY1 Deficiency is ~ 12 individuals born per year.

Gene therapy rescues disease phenotypes in NGLY1 rats.