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ION440

Oligonucleotide (Antisense oligonucleotide) · Gene silencing: Methyl CpG Binding Protein 2 (MECP2) · developed by IONIS PHARMACEUTICALS

Phase 1/2 Most advanced trial

IONS Developer

1 Registered trials

What is ION440?

ION440 is an antisense oligonucleotide targeting MECP2 mRNA, administered by intrathecal injection. It is being studied in MECP2 Duplication Syndrome, a neurological disorder caused by duplication of the MECP2 gene.

Summary written with AI from the sources listed at the end of this page.

At a glance

Drug type: Oligonucleotide (Antisense oligonucleotide)
Targets: Methyl CpG Binding Protein 2 (MECP2)
Mechanism: Gene silencing: Methyl CpG Binding Protein 2 (MECP2)
Therapy area: Neurology
Developer: IONIS PHARMACEUTICALS (IONS)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 1/2
UNII: 57WA6KDT3H

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT06430385	Phase 1/2	Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome	Recruiting	IONS

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

More from IONIS PHARMACEUTICALS

Donidalorsen FDA approved Olezarsen FDA approved Zilganersen FDA approved BIIB094 Phase 1 ION224 Phase 2 ION269 Phase 1 ION337 Phase 1/2 ION356 Phase 1 ION363 Phase 3 ION464 Phase 1 ION717 Phase 1/2 ION775 Phase 2

Sources

ClinicalTrials.gov NCT06430385

Updated Oct 3, 2026. Something wrong or missing? Report it on the IONS page.

Follow IONS, the company behind ION440

See IONS's upcoming FDA decisions, trial readouts, cash runway and dilution risk, updated every day from SEC, FDA and ClinicalTrials.gov filings.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z