Drug intelligence / Profile preview

AYX2

Development stage
Preclinical
Lead developer
Adynxx
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intrathecal
01

Overview

AYX2 is an experimental 18–base pair synthetic DNA decoy oligonucleotide designed to bind and inhibit the transcription factors Krüppel-like factor 6 (KLF6), Krüppel-like factor 9 (KLF9), and Krüppel-like factor 15 (KLF15), which are implicated in maintaining pathological gene expression underlying chronic pain states.[2][4] By sequestering these KLF transcription factors, AYX2 downregulates injury-induced transcriptional programs in dorsal root ganglia and spinal cord, leading to long-lasting analgesic effects in rat models of neuropathic and inflammatory pain after a single intrathecal administration.[1][2][4] It is under development as a non-opioid, disease‑modifying therapy for chronic pain, with preclinical pharmacokinetic and toxicology programs supported by NIH HEAL Initiative grants that describe AYX2 as a transcription factor decoy analgesic candidate.[3][6]

02

Targets

KLF9 (Krüppel-like factor 9)KLF6 (Krüppel-like factor 6)KLF15 (Krüppel-like factor 15)

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