GenomeRx is an RNA interference (RNAi) therapeutics company developing precisely engineered small interfering RNAs (siRNAs) to silence disease-driving genes in the liver and other tissues.
Our goal is simple: translate genetic insight into safe, durable, and accessible medicines for patients worldwide.
We are building a portfolio of siRNA medicines aimed at key regulators of lipid handling, inflammation, and steatosis, with the goal of addressing progressive liver diseases that currently lack effective, convenient treatments.
By targeting upstream drivers of fibrotic signaling, we aim to halt or slow organ damage in chronic kidney disease and related disorders before irreversible failure occurs.
Cardiometabolic disease sits at the intersection of dyslipidemia, insulin resistance, obesity, and chronic inflammation, ultimately driving cardiovascular events and organ damage. Our cardiometabolic programs focus on hepatic and systemic pathways that regulate lipid handling, glucose homeostasis, and inflammatory tone. By selectively silencing genes that exacerbate steatosis, atherogenic lipoprotein profiles, and metabolic stress, we aim to reduce long-term cardiovascular risk and improve outcomes for patients with conditions such as fatty liver disease, type 2 diabetes, and related cardiometabolic syndromes.
Where a single misbehaving gene can devastate a patient’s life, siRNA offers a direct path to intervention. GenomeRx is advancing early programs for genetically defined patient populations where silencing one gene could transform outcomes.
RNAi is a natural cellular process that uses short RNA strands to selectively silence specific genes. When harnessed as a medicine, synthetic siRNAs can be designed to:
This mechanism allows us to act before damage accumulates, offering a powerful complement to traditional small molecules and biologics that modulate proteins after they are made.
Chemically stabilized siRNA therapeutics
Ligand-directed administration if siRNA with a focus on liver targeting
Metabolic liver disease, fibrosis and kidney disease, and rare genetic indications
Preclinical, with multiple siRNA programs in discovery and lead optimization
Built to integrate computational design, high-throughput screening, and global development partners
All GenomeRx product candidates are investigational and have not been approved for use in any country.
We are designing an R&D engine that can go from in silico design to in vivo proof-of-concept rapidly, while maintaining the quality and documentation needed to support regulatory submissions and partnerships.