Pipeline

Anemia of inflammation is a common complication of chronic inflammatory conditions, malignancy, and chronic disease. It is driven by dysregulated iron homeostasis, primarily mediated through inappropriate activation of the hepcidin pathway, leading to restricted iron availability for erythropoiesis and subsequent drop in hemoglobin. Current treatment options are limited and often address downstream consequences rather than the underlying molecular drivers of disease.

DXO-1801 is a first-in-class GalNAc-conjugated liver-targeted siRNA therapeutic, designed to selectively silence a key gene within the hepcidin regulatory pathway. DXO-1801 aims to restore physiologic iron mobilization and improve iron availability for effective erythropoiesis.
The GalNAc delivery platform enables selective hepatocyte uptake and durable gene silencing with infrequent dosing, supporting a targeted and potentially well-tolerated approach. DXO-1801 is being developed to address the underlying biology of anemia of inflammation, with the goal of delivering meaningful and sustained correction of iron dysregulation.

Publications

November 3, 2025

Dxo-1801, a liver-targeted siRNA, attenuates inflammation-driven iron restriction and enhances erythropoiesis in non-human primates

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This program is a GalNAc-conjugated, liver-targeted siRNA therapeutic designed to selectively modulate expression of a select gene. The target and disease indication remain undisclosed at this stage.

The program leverages established RNA interference biology and hepatocyte-specific GalNAc delivery to enable precise and durable gene silencing in the liver. Development activities are supported by world-leading clinicians and scientific experts, ensuring strong translational relevance and alignment.

Early discovery and preclinical efforts in primates have generated highly encouraging results, supporting the underlying therapeutic hypothesis and continued advancement of the program. Further details regarding the target, indication, and development strategy will be disclosed as the program progresses.

This undisclosed delivery platform is designed to enable targeted and efficient delivery of siRNA to tissues beyond those addressable by existing liver-focused technologies. The platform aims to expand the therapeutic reach of RNA-based modalities while maintaining a strong emphasis on precision, durability, and translational relevance.