Drug intelligence / Profile preview

DXO-1801

Development stage
Preclinical
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
Subcutaneous
01

Overview

DXO-1801 is a fully chemically stabilized, liver-targeted, and highly specific GalNAC-conjugated small interfering RNA (siRNA). It functions by silencing the expression of a key gene within the hepcidin-regulatory pathway in hepatocytes. This mechanism is designed to attenuate inflammation-driven iron restriction, thereby enhancing iron availability and improving erythropoiesis. The drug is being developed as a potential therapeutic for anemia of inflammation, a complication associated with various chronic conditions such as inflammatory bowel and autoimmune diseases, chronic kidney disease, advanced solid tumors, and hematological malignancies.

02

Targets

TMPRSS6 (Transmembrane Serine Protease 6)

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