Drug intelligence / Profile preview

MECP2 AIMer

Development stage
Preclinical
Lead developer
Wave Life Sciences
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
01

Overview

**MECP2 AIMer** is an experimental RNA editing oligonucleotide designed to correct pathogenic *MECP2* nonsense mutations associated with Rett syndrome. It appears to belong to Wave Life Sciences' ADAR-mediated RNA editing platform, in which stereopure synthetic oligonucleotides called AIMers recruit endogenous adenosine deaminase acting on RNA to edit target transcripts at the RNA level rather than permanently altering DNA. In publicly available preclinical materials, MECP2 AIMers were used to restore MeCP2 protein expression in models carrying the **MECP2 R168X** mutation, supporting development as a precision medicine approach for genetically defined Rett syndrome patients. No marketed product name, clinical approval, or clear evidence of human clinical development for a specific MECP2 AIMer candidate was identified.

Other names
MECP2 RNA editing AIMerMECP-2 RNA editing AIMerMECP 2 RNA editing AIMer
02

Targets

MECP2 (Methyl-CpG-binding protein 2)

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