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APOB RNA editing oligonucleotide
- Development stage
- Preclinical
- Lead developer
- AIRNA Pharmaceuticals
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
APOB RNA editing oligonucleotide refers to a therapeutic candidate developed by AIRNA Bio using its RESTORE+ platform. This GalNAc-conjugated oligonucleotide is designed to recruit endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to perform precise Adenosine-to-Inosine (A-to-I) editing on the Apolipoprotein B (APOB) mRNA transcript. By introducing a specific protective variant identified through human genetics (UK Biobank), the therapy aims to lower LDL-cholesterol levels while avoiding the liver toxicity and steatosis associated with traditional APOB knockdown approaches (such as mipomersen). It is currently in preclinical development for the treatment of atherosclerotic cardiovascular disease (ASCVD).
- Other names
- APOB RNA editing oligoRESTORE+ APOB oligonucleotide
02
Targets
ADAR (Adenosine deaminases acting on RNA (ADAR) family)
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