Drug intelligence / Profile preview

NT-Z001

Development stage
Preclinical
Lead developer
Navega Therapeutics
Modality
Small Molecules, Gene Therapies
Administration
Intrathecal
01

Overview

NT-Z001 is a de-immunized epigenetic gene therapy developed by Navega Therapeutics for the non-opioid treatment of chronic pain. It targets a gain-of-function mutation in the SCN9A gene, which encodes the Nav1.7 voltage-gated sodium channel alpha subunit, implicated in inherited erythromelalgia, small fiber neuropathy, and other chronic debilitating pain disorders. The therapy uses an AI-enabled zinc-finger epigenome regulation platform to down-regulate Nav1.7 expression, thereby modulating pain signal transmission without opioid-related risks or addiction potential. It is delivered via an adeno-associated virus (AAV) vector (specifically AAV9). The drug is currently in preclinical development with plans for Phase I clinical trials.

Other names
NT Z001
02

Targets

SCN9A (Sodium channel protein type 9 subunit alpha)

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