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apolipoprotein E antisense oligonucleotide
- Development stage
- Unknown
- Lead developer
- AstraZeneca
- 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
Apolipoprotein E antisense oligonucleotide (specifically RO7486967 or ION449) is an investigational RNA-targeted therapy being developed by Roche and Genentech in collaboration with Ionis Pharmaceuticals. It is designed to treat Alzheimer's disease by reducing the production of apolipoprotein E (APOE) in the brain. APOE, particularly the APOE4 isoform, is the most significant genetic risk factor for late-onset Alzheimer's and is involved in the accumulation of amyloid-beta plaques and tau-related neurodegeneration. The drug utilizes antisense technology to bind to APOE mRNA, triggering its degradation and thereby lowering the levels of the APOE protein. It is currently being evaluated in Phase 1 clinical trials to assess its safety, tolerability, and pharmacokinetics in patients with Alzheimer's disease.
- Other names
- apolipoprotein E antisense oligonucleotideAPOE-directed antisense oligonucleotideAPOE-targeted ASO
02
Targets
APOE (Apolipoprotein E)
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