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OCCT-lynx1 siRNA is a preclinical-stage therapeutic candidate developed by Ophidion for the treatment of cognitive impairment associated with neurodegenerative diseases such as Alzheimer's and Parkinson's disease. The drug utilizes Ophidion's proprietary CNS Carrier Technology (OCCT) platform, which employs a nanoparticle-based "Trojan horse" strategy to deliver small interfering RNA (siRNA) across the blood-brain barrier via intravenous injection. The active ingredient is an siRNA designed to knock down the expression of Lynx1, a membrane-tethered protein that binds to and modulates nicotinic acetylcholine receptors (nAChRs). Lynx1 normally acts as a molecular brake on synaptic plasticity, suppressing memory and learning. By silencing Lynx1 expression, the therapy aims to enhance cholinergic signaling and restore cognitive function. Preclinical studies in mouse models have demonstrated successful target knockdown and improved novel object recognition memory following administration.
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