Drug intelligence / Profile preview

ION581

Development stage
Preclinical
Lead developer
Ionis Pharmaceuticals
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intrathecal
01

Overview

ION581 appears to be an investigational **antisense oligonucleotide** program from Ionis directed at Angelman syndrome biology, but the public evidence available for the exact code name is sparse and not well corroborated across primary sources. Secondary pipeline databases describe ION-581 as an RNA-targeted candidate intended to increase **UBE3A** expression by inhibiting pathogenic transcription related to Angelman syndrome. However, Ionis's currently visible public Angelman program is more consistently described under the closely related code **ION582**, an intrathecally administered antisense oligonucleotide in clinical development. Because the exact identity and status of ION581 are not clearly confirmed in authoritative public primary sources, the characterization of ION581 itself should be treated as uncertain.

02

Targets

UBE3A-ATS (UBE3A antisense transcript)

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