Drug intelligence / Profile preview

RN276

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
Administration
Intravenous, Subcutaneous
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Overview

RN276 is a preclinical small interfering RNA (siRNA) therapeutic candidate developed by Shanghai Rona Therapeutics for the treatment of Alzheimer's disease. It is designed to target the mRNA of the Microtubule-associated protein tau (MAPT), thereby silencing the expression of the Tau protein, which is a central driver of neurodegeneration when it aggregates into neurofibrillary tangles. RN276 utilizes Rona's proprietary APOLLO platform, a delivery technology engineered for extra-hepatic applications that is specifically designed to cross the blood-brain barrier (BBB). This allows the siRNA to reach the central nervous system following systemic administration, offering a potential disease-modifying approach for tauopathies by reducing the overall burden of pathological Tau in the brain.

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Targets

MAPT (Microtubule-associated protein tau)

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