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RN201

Development stage: Preclinical
Lead developer: Rona Therapeutics
Modality: Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics

Overview

RN201 is a preclinical RNA-based therapeutic candidate developed by Shanghai Rona Therapeutics for the treatment of epilepsy. It is built upon the company's proprietary APOLLO platform, which is designed for the delivery of oligonucleotide therapeutics to extra-hepatic tissues, specifically targeting the central nervous system (CNS) for neurological indications. As an siRNA-based therapy, RN201 is intended to silence a specific gene involved in the pathogenesis of epilepsy to reduce seizure activity, although its exact molecular target remains undisclosed by the developer.

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