Rgenix is a privately-held clinical-stage biopharmaceutical company developing first-in-class drugs that target key pathways in cancer progression.

310 E 67th St, Suite 1-12‎ New York, NY
(646) 856-9261

Our Science

RNA-DRIVEr™ is a clinically validated oncology discovery platform

The vast majority of cancer deaths are caused by metastatic tumors, yet currently approved cancer drugs do not by design target the biology of metastatic disease. As a result, most advanced cancer patients succumb to illness despite multiple treatments. If we could specifically target the genes that drive the growth of metastatic disease, we could positively impact patient survival and ultimately achieve curative outcomes. This is our primary goal at Inspirna.



1. We discover critical targets that drive the growth of metastatic disease.

We start by purifying the extremely rare aggressive cancer cells from multiple patients’ tumors that are highly effective in driving the growth of metastatic disease. We then search for a protein that is similarly altered in the highly aggressive cancer cells of all patients studied. Unlike conventional proteomic approaches, our RNA-DRIVEr™ (RNA dysregulated Drug Target In Vivo Elucidation) approach allows us to survey all ~20,000 human proteins in order to identify the causative driver gene while avoiding correlative protein changes often observed in tumor tissue.

2. We test and validate the role of the discovered gene in driving metastatic disease.

We perform a series of molecular and genetic studies to determine the impact of the identified gene on driving the growth of metastatic disease in mice, conducting extensive studies in state-of-the-art models including cell line and patient derived xenografts using immunocompetent and immunocompromised mouse models. These mouse studies are corroborated by clinical association studies where the expression levels of the gene are tested for association with cancer patients’ survival outcomes in multiple clinical datasets. These animal studies are complemented by Inspirna’s in vitro phenotypic assays that mechanistically define the mechanism of action and establish robust systems for subsequent high-throughput screens.

3. Our experienced team develops new drug candidates suitable for clinical evaluation.

Having developed robust in vitro and in vivo phenotypic assays as well as genetically modified cells and models, we screen for lead small-molecules or monoclonal antibodies that specifically alter the activity of the critical protein. Such leads are optimized to yield highly potent first in class therapeutics that exhibit strong activity against both primary tumor growth and metastatic disease progression in vivo.

4. We then transform cancer medicine by leveraging RNA biology driven insights.

Inspirna’s deep mechanistic studies of the critical gene across a series of in vivo and in vitro models, coupled with our human clinicopathologic correlative data and our highly experienced clinical team uniquely positions Inspirna to advance first-in-class drug candidates from IND to clinical proof of concept studies and beyond. Our extensive mechanistic pre-clinical studies guide our biomarker enriched patient selection, which is validated and refined by phase 1b expansion trial findings. Since the critical driver genes we have thus far identified drive metastatic disease progression by a large fraction of cancer subtypes, the potential for major impact on the cancer population at large is high.