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ION356

Gene therapy (AAV vector) · Gene replacement: PLP1 · developed by IONIS PHARMACEUTICALS

Phase 1 Most advanced trial

IONS Developer

1 Registered trials

What is ION356?

ION356 is an AAV gene therapy administered by intrathecal injection for Pelizaeus-Merzbacher Disease, a genetic neurological disorder affecting myelin formation.

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

At a glance

Drug type: Gene therapy (AAV vector)
Targets: PLP1
Mechanism: Gene replacement: PLP1
Therapy area: Neurology
Developer: IONIS PHARMACEUTICALS (IONS)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 1

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT06150716	Phase 1	Pelizaeus-Merzbacher Disease	Recruiting	IONS

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

Conditions studied

Pelizaeus-Merzbacher Disease

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 ION363 Phase 3 ION440 Phase 1/2 ION464 Phase 1 ION717 Phase 1/2 ION775 Phase 2

Sources

ClinicalTrials.gov NCT06150716

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

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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