Drug intelligence / Profile preview

RESTORE+

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

RESTORE+ is a GalNAc-conjugated RNA editing platform developed by AIRNA Bio that utilizes endogenous Adenosine Deaminase Acting on RNA (ADAR) to achieve precise, reversible, and programmable A-to-I (adenosine-to-inosine) edits at the RNA level. The platform is designed to introduce genetically validated gain-of-function or protective variants into target transcripts to treat diseases with high unmet needs, such as atherosclerotic cardiovascular disease (ASCVD). In preclinical studies, RESTORE+ oligonucleotides have been used to introduce a single A-to-G edit in the 3'UTR of the Low Density Lipoprotein Receptor (LDLR) to increase its expression, as well as to introduce protective variants in the Apolipoprotein B (APOB) transcript. This approach aims to provide a safer and more effective alternative to traditional loss-of-function therapies by directly enhancing protein function or expression.

Brand names
RESTORE+
02

Targets

ASGPR (Asialoglycoprotein Receptor 1)ADAR1/ADAR2 (Adenosine deaminase acting on RNA 1 and 2)Alpha-1 antitrypsin PiZ-mutant messenger RNA (SERPINA1 PiZ mRNA)

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