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zilganersen
- Development stage
- Approved
- Lead developer
- Ionis Pharmaceuticals
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
**Zilganersen** is a glial fibrillary acidic protein-directed antisense oligonucleotide developed by Ionis Pharmaceuticals for Alexander disease. It binds glial fibrillary acidic protein messenger RNA and reduces production of glial fibrillary acidic protein, whose pathological overproduction and accumulation in astrocytes drives Alexander disease caused by GFAP variants. Zilganersen is administered as a quarterly intrathecal injection and was approved in the United States in September 2026 under the brand name Zanvastro for pediatric and adult patients with Alexander disease.
- Brand names
- Zanvastro
- Other names
- 2'-o-(2-methoxyethyl)-d-ribose antisense oligonucleotide targeting glial fibrillary acidic protein mrna2'-O-methoxyethyl antisense oligonucleotide targeting GFAP mRNA
02
Targets
Glial fibrillary acidic protein messenger RNA
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