Company intelligence / Profile preview
Grace Science, LLC
Menlo Park, California, United Statesgracescience.com ↗
- Ownership
- Private
- Employees
- ~50
- Development stage
- Phase 1
01
Overview
Grace Science, LLC has initiated a Phase 1/2/3 open-label clinical trial of GS–100, an AAV9 gene replacement therapy for NGLY1 Deficiency. The first two patients have been treated with no drug-related adverse events reported so far. The company’s deep expertise in the NGLY1 pathway positions it to expand into oncology and neurodegeneration. GS–100 has received Orphan Drug Designation from both FDA and EMA, Rare Pediatric Disease Designation from FDA, and Fast Track Designation from FDA[4][5].
- Therapeutic areas
- OncologyRare DiseasesNeurology
- Modalities
- AAV-based gene therapySmall molecule drugs
- Industry
- Biotech
02
Drug pipeline
3 assets2 more assets in the full pipeline.
Request access →03
Partnerships
Takeda Pharmaceuticals (collaboration on animal models)[3]Andelyn Biosciences (manufacturing partner for GS–100)[7]
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