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ION269

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

Overview

ION269 is an investigational antisense oligonucleotide (ASO) medicine designed to lower the amount of amyloid precursor protein (APP) for the potential treatment of Alzheimer's disease. It is initially being studied in adults with Down syndrome who have a genetic risk of developing Alzheimer's disease. The drug works by targeting and reducing APP, which is implicated in the pathogenesis of Alzheimer's disease. ION269 is being developed by Ionis Pharmaceuticals and is currently in Phase 1 clinical trials, specifically recruiting adults with Down syndrome[1][2][3][4][5].

Targets

Aβ (Amyloid-beta peptides and aggregates)

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