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LDLR RNA editing oligonucleotide
- Development stage
- Preclinical
- Lead developer
- AIRNA Bio
- 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
LDLR RNA editing oligonucleotide is a therapeutic candidate developed by AIRNA Bio designed to treat atherosclerotic cardiovascular disease (ASCVD) by increasing the expression of the Low Density Lipoprotein Receptor (LDLR). Utilizing the RESTORE+ platform, the drug consists of a GalNAc-conjugated oligonucleotide that recruits endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to perform a precise Adenosine-to-Inosine (A-to-I) edit in the 3'UTR of the LDLR mRNA. This edit functionally replicates a protective gain-of-function genetic variant found in the Icelandic population, which leads to increased LDLR protein expression, thereby enhancing the clearance of LDL cholesterol from the blood. Preclinical studies have demonstrated significant increases in LDLR expression and substantial reductions in LDL-C levels in animal models.
- Other names
- LDLR RNA editing oligo
02
Targets
ADAR (Adenosine deaminases acting on RNA (ADAR) family)Low-density lipoprotein receptor pre-messenger RNA (LDLR pre-mRNA)
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