Our Science Platform

Our Science:
Precisely Engineered siRNA Medicines

GenomeRx is building a modular platform to design, optimize, and advance siRNA drug candidates that silence disease-driving genes with high potency and durability, while minimizing off-target effects.

How siRNA
Therapeutics Work

Every cell uses mRNA as a set of instructions for building proteins. In many diseases, a single gene is overactive, mis-regulated, or produces a toxic protein.

siRNA therapeutics are short pieces of RNA designed to:

  • Match a specific mRNA sequence from a disease-related gene
  • Guide the cell’s own RNAi machinery to that mRNA
  • Cut and degrade the mRNA so the protein is never produced in the first place

Because the mechanism is upstream of protein production, small changes in mRNA levels can translate into meaningful reductions in disease-driving proteins.

The GenomeRx siRNA Design Philosophy

We focus on three pillars:

Smart Target
Selection

  • Prioritize genes with human genetic validation and strong mechanistic rationale
  • Focus on pathways where partial, controlled knockdown is likely to be therapeutic rather than toxic
  • Emphasize indications where RNAi can confer clear advantages over existing modalities

Chemically Stabilized siRNAs

  • Use fully modified backbones and sugars to improve stability in circulation and within tissues
  • Engineer strands for high potency, with a bias toward on-target activity and reduced off-target risk
  • Optimize designs for durable gene silencing that supports infrequent dosing

Ligand-Directed
Delivery

  • Deploy conjugates that naturally home to target tissues, initially, the liver
  • Tune chemistry and formulation for reproducible, scalable manufacturing
  • Maintain flexibility to expand to additional tissues as new ligands and delivery strategies mature

A Platform Built Around Translational Questions

Rather than treating “platform” and “pipeline” as separate activities, GenomeRx designs its experimental plan to answer the questions that matter most for clinical translation:

  • Can we reach the right cells at clinically relevant doses?
  • How deep and durable is gene knockdown in vivo?
  • What biomarkers best represent pharmacodynamic effect and safety?
  • How do we design early studies that de-risk later stages?

This approach drives a common workflow across programs:

In Silico Design & Prioritization

Sequence selection, off-target prediction, and manufacturability assessment

In Vitro Screening

Potency, specificity, and safety assessments across multiple cell types

In Vivo Proof-of-Concept

Pharmacokinetics, pharmacodynamics (target mRNA/protein reduction), and preliminary safety

Clinical-Readiness Planning

CMC strategies, biomarker selection, and early regulatory engagement

Patient-Centric by Design

Although GenomeRx is an early-stage company, we are designing our medicines and our platform from the patient’s perspective:

  • Infrequent dosing is a core goal, wherever feasible
  • Subcutaneous administration is preferred over more invasive routes
  • Global access shapes our thinking on manufacturability and cost from day one
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