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Grace Science

Private
#NGLY1 Deficiency#GM3 Synthase Deficiency#Ultra-Rare Neurological Disease#Gene Therapy#AAV-Based Therapeutics

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

Sector:Biotechnology / Gene Therapy (Ultra-Rare Disease)
Headquarters:Waltham, Massachusetts, USA
Founded:2017
Website:www.gracescience.com/
Funding Round:Series A and venture-backed (amounts undisclosed)
Total Funding:Undisclosed venture-backed funding

Funding & Investor Relations

IPO Outlook / Plans

None publicly disclosed; private ultra-rare-disease gene therapy developer.

Upcoming Milestones & Key Dates

H2 2026
Regulatory

FDA Type B meeting on GS-101 manufacturing and clinical path forward

Late 2026
Regulatory

Potential IND filing for GS-101 (NGLY1) if CMC requirements resolved

Clinical Development Pipeline

GS-101 (NGLY1 gene therapy)

Target: AAV-mediated NGLY1 gene replacement
NGLY1 Deficiency (ultra-rare congenital disorder of deglycosylation)
Preclinical
Phase 1
Phase 2
Phase 3
Approved

GS-201 (ST3GAL5 gene therapy)

Target: AAV-mediated ST3GAL5 gene replacement
GM3 Synthase Deficiency (salt-and-pepper syndrome, ultra-rare)
Preclinical
Phase 1
Phase 2
Phase 3
Approved

GS-Discovery

Target: AAV gene therapy platform
Additional ultra-rare neurological diseases (undisclosed)
Preclinical
Phase 1
Phase 2
Phase 3
Approved

Recent News

Leadership Team

Ken Drazan

Founder and Chief Executive Officer

Careers & Job Openings

See all open roles

Multiple Departments
See careers page
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