Companies in #NGLY1 Deficiency

Grace Science is a clinical-stage biotechnology company developing gene therapies for ultra-rare neurological diseases, including NGLY1 Deficiency and GM3 Synthase Deficiency. The company is led by founder and CEO Ken Drazan and is built around a parent-driven drug development model, with its lead gene therapy program targeting a disease that affects the founder's own daughter and a small global patient population.