Drug intelligence / Profile preview

RN508

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
01

Overview

RN508 is a preclinical-stage small interfering RNA (siRNA) candidate developed by Shanghai Rona Therapeutics. It is part of the company's proprietary APOLLO platform, which is specifically engineered for the delivery of RNA therapeutics to extra-hepatic tissues, with a focus on the central nervous system (CNS). RN508 utilizes the RNA interference (RNAi) pathway to achieve potent and durable silencing of a specific, currently undisclosed gene associated with neurological or neurodegenerative disorders. By employing advanced chemical modifications and specialized delivery ligands, RN508 aims to overcome the challenges of systemic delivery to the brain and spinal cord, representing a key expansion of Rona's pipeline beyond liver-targeted indications.

02

Targets

INHBE (Inhibin beta E chain)

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