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.
Company Info
Funding & Investor Relations
None publicly disclosed; private ultra-rare-disease gene therapy developer.
Upcoming Milestones & Key Dates
FDA Type B meeting on GS-101 manufacturing and clinical path forward
Potential IND filing for GS-101 (NGLY1) if CMC requirements resolved
Clinical Development Pipeline
Recent News
Grace Science, a small biotech developing an ultra-rare-disease gene therapy, says an FDA request for additional manufacturing data is delaying treatment for the founder's own daughter and other patients with no therapeutic alternative.
Leadership Team
Founder and Chief Executive Officer
Careers & Job Openings