GenomeRx, Inc. is an early-stage biotechnology company developing next-generation precision RNAi therapeutics. We are building a modern siRNA engine that silences the genes driving disease. We focus on targets with strong human genetic validation, which strengthens confidence in each program and guides us toward precise, durable treatments.
Our pipeline strategy covers three areas where RNAi can make a real difference: neurological disorders of the CNS, retinal diseases, and liver-mediated metabolic and cardiometabolic conditions. Across all three, our work rests on rigorous science, careful siRNA design, and disciplined target selection.
We are committed to global access, bringing precision RNAi medicines to patients worldwide.
To develop best-in-class siRNA therapeutics that silence disease-causing genes with high potency, durability, and safety – addressing significant unmet needs in Parkinson’s disease (CNS), dry AMD, and liver metabolic/cardiometabolic diseases.
We start with strong human biology and genetics, then design programs that are mechanistically grounded and clinically meaningful.
Each new program is chosen not only for its therapeutic promise, but also for what it teaches us about siRNA design, delivery, and translation across indications.
GenomeRx is built to take advantage of a distributed model—leveraging best-in-class partners for specialized functions while maintaining a lean internal core focused on design, data, and strategy.
RNA therapeutics leader and U.S. patent attorney with 20+ years of experience in siRNA design, nucleic acid chemistry, targeted delivery, drug development, and IP strategy.
Experts in RNA chemistry, liver disease, nephrology, and translational pharmacology.
Seasoned industry leaders guiding clinical development, regulatory strategy, and global market access.
Decisions are grounded in data, reproducible experiments, and honest interpretation.
We design processes to move quickly without cutting scientific or ethical corners.
With our partners and stakeholders, we’re clear about risk, timelines, and what we know—or don’t yet know
From early on, we consider manufacturing, cost, and distribution challenges that influence who can ultimately receive the medicines we develop.
GenomeRx seeks collaborations across the drug development continuum, including:
We engage flexibly—through research collaborations, option-to-license agreements, or joint development structures—based on the needs of each program.