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ION464
- Development stage
- Phase 1
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Small Molecules, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
ION464 is an investigational antisense oligonucleotide designed to target and reduce the production of alpha-synuclein protein by binding to its mRNA. Alpha-synuclein accumulation is implicated in the pathogenesis of neurodegenerative disorders such as Parkinson's disease and multiple system atrophy (MSA). By inhibiting alpha-synuclein synthesis, ION464 aims to prevent or slow neurodegeneration associated with these synucleinopathies. The drug is administered via intrathecal injection and has shown efficacy in preclinical models by reducing alpha-synuclein levels and pathology. It is being developed by Ionis Pharmaceuticals in collaboration with Biogen[1][2][3][4].
- Other names
- BIIB101BIIB-101BIIB 101IONIS-BIIB6RxIONIS-BIIB-6RxIONIS-BIIB 6Rx
02
Targets
Alpha-synuclein messenger RNA
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