Drug intelligence / Profile preview

ASO-18

Development stage
Preclinical
Lead developer
University of South Florida
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intracerebroventricular
01

Overview

ASO-18 is a cross-species antisense oligonucleotide (ASO) GapmeR designed to target and knock down the expression of FK506-binding protein 51 (FKBP51), a co-chaperone of the 90kDa heat shock protein (Hsp90). Developed by researchers at the University of South Florida, ASO-18 targets conserved regions of the human *FKBP5* and mouse *Fkbp5* genes. FKBP51 has been implicated in stabilizing neurotoxic tau species in tauopathies such as Alzheimer's disease. In preclinical studies, ASO-18 demonstrated high potency and selectivity, reducing FKBP51 levels without affecting the close homolog FKBP52. Administration of ASO-18 in neuronal models and tau transgenic mice led to significant reductions in total tau and phospho-tau levels, suggesting potential therapeutic utility for tauopathies and related neuropsychiatric disorders.

02

Targets

FKBP5 (FK506-binding protein 5)

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