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Drug intelligence / Profile preview
RTX-7340
- Development stage
- Preclinical
- Lead developer
- Rumi Therapeutics
- Modality
- Small Molecules
01
Overview
RTX-7340 (also known as ENT-7340) is an investigational, IND-ready small-molecule inhibitor of phosphodiesterase 2A (PDE2A). Originally discovered by Enthorin Therapeutics, the drug is being developed by Rumi Therapeutics under an option-to-license agreement for the treatment of Fragile X syndrome (FXS) and other neurodevelopmental disorders. By selectively inhibiting PDE2A, RTX-7340 modulates intracellular cyclic adenosine monophosphate (cAMP) and cyclic guanosine monophosphate (cGMP) signaling pathways, which are critical for neuronal function and synaptic plasticity. Preclinical studies in Fmr1-knockout mouse models of Fragile X syndrome have demonstrated that PDE2 inhibition corrects disease-associated electroencephalography (EEG) abnormalities and normalizes cognitive and autism-relevant behavioral deficits.
02
Targets
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