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ETX301
- Development stage
- Preclinical
- Lead developer
- Encoded Therapeutics
- Modality
- Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Intrathecal
01
Overview
ETX301 is an investigational AAV9-based vectorized microRNA (miRNA) therapy being developed by Encoded Therapeutics for the treatment of chronic neuropathic pain, specifically targeting lower limb post-amputation neuroma. The drug is designed to selectively and durably knock down the expression of the SCN9A gene, which encodes the NaV1.7 voltage-gated sodium channel, a key mediator of pain signaling in nociceptors. By targeting dorsal root ganglia (DRG) nociceptive neurons via a single intrathecal injection, ETX301 aims to provide long-term therapeutic benefit. The program was nominated as a development candidate in May 2026, with an Investigational New Drug (IND) application anticipated in 2027.
02
Targets
SCN9A (Sodium channel protein type 9 subunit alpha)
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