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ST-503
- Development stage
- Unknown
- Lead developer
- Sangamo Therapeutics
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Classical Binding Small Molecules → Small Molecules, Gene Silencing → Gene Therapies, Covalent Small Molecules → Small Molecules, Zinc Finger Nucleases → Other Nucleases → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intrathecal
01
Overview
ST-503 is an investigational epigenetic gene therapy developed by Sangamo Therapeutics for the treatment of chronic neuropathic pain associated with idiopathic small fiber neuropathy (iSFN). It employs an adeno-associated virus (AAV) vector to deliver a zinc finger repressor (ZFR) that selectively silences the SCN9A gene, which encodes the Nav1.7 sodium channel—a key mediator in pain signaling pathways. By repressing Nav1.7 expression in sensory neurons, ST-503 aims to reduce refractory pain that is resistant to conventional therapies such as antidepressants, anticonvulsants, and opioids. Preclinical studies have shown significant reduction in pain hypersensitivity and good tolerability with no off-target effects observed in nonhuman primates. The drug is administered via a single intrathecal injection and is currently entering Phase 1/2 clinical trials for iSFN[1][3][5][6].
- Other names
- Nav1.7 zinc finger repressorNav-1.7 zinc finger repressorNav 1.7 zinc finger repressorSCN9A zinc finger repressorSCN-9A zinc finger repressorSCN 9A zinc finger repressorAAV-ZFR-Nav1.7AAV-ZFR-Nav-1.7AAV-ZFR-Nav 1.7
02
Targets
SCN9A (Sodium channel protein type 9 subunit alpha)
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