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.

This program is a GalNAc-conjugated, liver-targeted siRNA therapeutic designed to selectively modulate expression of a gene associated with beneficial metabolic outcomes in preclinical models and human genetic studies.
The program aims to address key features of metabolic syndrome through targeted hepatic gene silencing and downstream modulation of pathways involved in systemic metabolic homeostasis.

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Leveraging established RNA interference biology and hepatocyte-specific GalNAc delivery, the program is designed to enable precise and durable modulation of pathways relevant to metabolic health. Development activities are supported by leading clinicians and world-renowned scientific experts to ensure strong translational relevance and clinical alignment.

Early discovery and preclinical studies in a diet-induced obesity non-human primate model have generated highly encouraging results, supporting the 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 program is a GalNAc-conjugated, liver-directed siRNA therapeutic in discovery-stage development for the treatment of CKD-associated anemia, a serious and highly prevalent complication of chronic kidney disease that contributes substantially to morbidity, cardiovascular complications, and impaired quality of life.

Although current treatment options, including erythropoiesis-stimulating agents (ESAs), have improved disease management, their use may be limited by safety concerns, variable patient responsiveness, and the challenges associated with long-term anemia control in CKD populations.

The program is designed to selectively regulate expression of a target gene involved in pathways governing erythropoiesis, iron homeostasis, and renal disease-associated hematologic dysfunction. By leveraging RNA interference technology and hepatocyte-specific GalNAc delivery, the therapeutic approach aims to achieve durable and tissue-selective modulation of disease relevant biology with the potential to improve hematologic outcomes while addressing limitations of existing therapies.Additional details regarding the target, development strategy, and clinical positioning will be disclosed as the program progresses.