Zilganersen

DB18161ApprovedInvestigationalGene Therapies

Zilganersen is a GFAP-directed antisense oligonucleotide indicated for Alexander disease in pediatric and adult patients, administered intrathecally to reduce synthesis of glial fibrillary acidic protein. Alexander disease is caused by variants in the GFAP gene that lead to overproduction and toxic accumulation of GFAP in astrocytes, and dysfunction in astrocytes can over time damage neurons and myelin.

Mechanism: Zilganersen is an antisense oligonucleotide that binds glial fibrillary acidic protein (GFAP) pre-mRNA. This binding causes degradation of the GFAP pre-mRNA, which results in a reduction of GFAP protein synthesis. Alexander disease is caused by variants in the GFAP gene that lead to overproduction and toxic accumulation of GFAP in astrocytes.
Curator reviewed · 3 references
Primary indication: Zilganersen is indicated for the treatment of Alexander disease in pediatric and adult patients.Curator reviewed · 1 structured indication
Code names: ION-1166998 FREE ACID · ION-373

Resolves to

Compound

UNIIAXQ9493NT2CAS2305355-56-8

Targets

P14136GFAP

What you can answer from here — as of September 11, 2026

2Protein targetsEach mapped to UniProt, with action and pharmacological action

Listed above

2Clinical trialsPhase, status and sponsor resolved per trial

1Structured indicationCondition, population, route and combination as fields, not prose

4+ReferencesStructured and connected to the statements they support

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