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ION440

Development stage: Phase 2
Lead developer: Ionis Pharmaceuticals
Modality: 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

Overview

ION440 is an investigational antisense oligonucleotide (ASO) drug developed by Ionis Pharmaceuticals for the treatment of MECP2 Duplication Syndrome (MDS), a rare genetic disorder characterized by developmental delays, impaired motor function, and seizures. The drug is designed to decrease production of the MECP2 (methyl CpG binding protein 2) protein. It is administered intrathecally (directly into the spinal canal). As of 2024, ION440 is in Phase 1/2 clinical trials evaluating its safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with MDS.

Targets

Methyl-CpG binding protein 2 messenger RNA (MECP2 mRNA)

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