Drug intelligence / Profile preview

CITY-RBP4

Development stage
Preclinical
Lead developer
City 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

CITY-RBP4 is a next-generation RNA interference (RNAi) therapeutic candidate being developed by City Therapeutics for the treatment of Stargardt disease. The drug utilizes a small interfering RNA (siRNA) to target and knockdown the expression of retinol binding protein 4 (RBP4) mRNA in the liver. RBP4 is a liver-produced protein responsible for transporting retinol (vitamin A) to the eye; in Stargardt disease, excessive retinoid accumulation leads to the formation of toxic byproducts that cause progressive vision loss. By reducing systemic RBP4 levels, CITY-RBP4 aims to lower the delivery of retinol to the retina, thereby mitigating pathogenic retinoid-related processes. The program is currently in the IND-enabling phase of development.

02

Targets

RBP4 (Retinol-binding protein 4)

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