ATL-301

ATL-301 is a preclinical-stage divalent small interfering RNA (di-siRNA) therapeutic developed by Atalanta Therapeutics for the treatment of various pain conditions. The drug utilizes a proprietary dicer-substrate siRNA platform to target and silence the *SCN9A* gene, which encodes the Nav1.7 voltage-gated sodium channel, a protein essential for pain signaling in sensory neurons. By leveraging RNA interference, ATL-301 reduces Nav1.7 mRNA expression and eliminates Nav1.7 currents with high potency and selectivity, avoiding off-target effects on other sodium channel paralogs. Preclinical rodent studies have shown that a single intrathecal administration of ATL-301 provides dose-dependent analgesia lasting approximately three months, demonstrating a long duration of action and broad distribution throughout the central nervous system.

Other names

NAV 1.7 (ATL-301)SCN9A di-siRNASCN-9A di-siRNASCN 9A di-siRNA