Posted by Michael Wonder on 15 Apr 2026
US FDA grants RMAT designation to GS-100, Grace Science’s gene therapy to treat NGLY1 deficiency
14 April 2026 - Grace Science announced today that the US FDA has granted regenerative medicine advanced therapy 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.
