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GEM 00220
- Development stage
- Unknown
- Lead developer
- Northern Therapeutics
- Modality
- Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
GEM 00220 is an allogeneic mesenchymal stem/stromal cell (MSC) therapy that has been genetically modified to overexpress human endothelial nitric oxide synthase (eNOS). Developed by Northern Therapeutics, the therapy is designed to treat septic shock by leveraging the innate immunomodulatory properties of MSCs alongside the vasoprotective and anti-inflammatory effects of nitric oxide. In sepsis, the localized production of nitric oxide by these enhanced cells is intended to restore vascular integrity, prevent microvascular thrombosis, and modulate the systemic hyper-inflammatory response that leads to multi-organ failure. GEM 00220 is currently being evaluated in the Phase 1 AMETHYST trial to determine its safety and tolerability in patients with septic shock.
- Other names
- Genetically enhanced mesenchymal stem cellseNOS-enhanced MSCsAdvanced Mesenchymal Enhanced Cell Therapy
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