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N-acetyl cysteine dendrimer conjugate
- Development stage
- Phase 1
- Lead developer
- Ashvattha Therapeutics
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, Polymer-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
OP-101 is an investigational anti-inflammatory and antioxidant drug designed as a covalent conjugate of generation-4 hydroxyl-terminated polyamidoamine (PAMAM) dendrimer and N-acetyl cysteine (NAC), linked via a disulfide bond. The drug is engineered to selectively target activated macrophages and microglia in inflamed tissues, including the brain. Upon cellular uptake by these reactive immune cells, the disulfide bond is cleaved intracellularly by glutathione, releasing NAC directly at sites of inflammation. This targeted delivery enhances anti-inflammatory and antioxidant effects while minimizing systemic exposure. OP-101 has been developed primarily for severe inflammatory conditions such as adrenoleukodystrophy (ALD) and severe COVID-19, where it aims to reduce neuroinflammation, oxidative stress, cytokine storm, lung injury, multi-organ failure, and neuronal injury[2][3][4][5][6][8]. The developer is Orpheris (a subsidiary of Ashvattha Therapeutics).
- Other names
- OP-101OP101OP 101n-acetyl cysteine dendrimer conjugate
02
Targets
NF-κBIKBKB (IKKβ)
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