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DiR-FXS
- Development stage
- Preclinical
- Lead developer
- Aptadir Therapeutics
- Modality
- RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
DiR-FXS is a preclinical-stage RNA therapeutic being developed by Aptadir Therapeutics for the treatment of Fragile X Syndrome (FXS). It utilizes a novel modality known as DNMT1-interacting RNA (DiR). The mechanism involves a locus-specific RNA that targets the *FMR1* gene locus. By interacting with or inhibiting DNA methyltransferase 1 (DNMT1) specifically at this site, DiR-FXS aims to prevent or reverse the aberrant DNA methylation that typically silences the *FMR1* gene in patients with FXS, thereby restoring the production of the FMRP protein.
02
Targets
DNMT1 (DNA (cytosine-5)-methyltransferase 1)
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