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movronersen
- Development stage
- Unknown
- Lead developer
- Ionis Pharmaceuticals
- Modality
- 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
- Administration
- Intrathecal
01
Overview
**Movronersen** is an investigational, intrathecally administered antisense oligonucleotide developed by Ionis Pharmaceuticals, with Biogen previously serving as a development partner. It is designed to bind and promote degradation of **SNCA messenger RNA**, thereby reducing production of alpha-synuclein, a pathogenic protein that accumulates in glial cytoplasmic inclusions in multiple system atrophy. Movronersen is being evaluated in the HORIZON clinical study in adults with multiple system atrophy as a potential disease-modifying RNA-targeted therapy.
- Other names
- ION-464ION464ION 464BIIB-101BIIB101BIIB 101
02
Targets
Alpha-synuclein messenger RNA
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