Inspirna’s novel target discovery platform, miRNA-DRIVEr, reveals new cancer drivers through microRNA profiling.
Inspirna has developed the unique expertise to discover novel and actionable cancer drivers that affect large patient populations. We conduct comprehensive studies including genomic, transciptomic, proteomic and metabolomic approaches for the identification and validation of our targets as well as the characterization of the underlying biology. The team has extensive expertise across all areas of drug development to move programs quickly from lab-to-clinic. Our approach has generated extensive scientific and preclinical data, which have led to multiple publications in high profile journals.
Our proprietary and clinically-validated miRNA-DRIVEr platform, coupled with our expertise in miRNA mapping, allows us to reveal new cancer drivers and actionable targets of miRNA dysregulation.
miRNA-DRIVEr = miRNA Dysregulated Drug Target In Vivo Elucidation
Why target miRNA regulated pathways?
Micro RNAs (miRNAs)* have important biological functions:
- Regulate gene expression at the post-transcriptional level;
- Modulate cell growth and differentiation;
- Regulate pathways and networks via coordinated activities;
- Act co-operatively with other miRNAs and with transcription factors, which are frequent targets of miRNAs.
miRNA dysregulation is a frequent contributor to cancer growth and progression.
- Overexpression or depletion of distinct sets of miRNAs is frequently found in cancer.
- Delineating miRNA functional effects requires elucidation of their upstream regulators and downstream targets.
*short, endogenous noncoding RNAs 19–22 nucleotides long
Our miRNA-DRIVEr platform reveals actionable miRNA-dysregulated cancer targets
We are dedicated to stopping metastatic progression and providing long-term survival benefits for patients by providing more practical treatment options that prolong survival without decreasing quality of life. Our innovative medicines possess improved dosing schedules and safety profiles that will become seamless and simple additions to the existing standard of care (i.e., oral BID) to treat indications with large patient populations and high unmet need.
DISCOVER
We discover critical targets that drive the growth of metastatic disease.
We start by purifying the extremely rare, aggressive cancer cells that are highly effective in driving the growth of metastatic disease. We then identify miRNAs that are dysregulated in these metastatic cells and search for the protein that is regulated by them.
microRNA Profiling
Generic Parental Cell
Poorly metastatic
Inspirna’s Proprietary
miRNA-DRIVEr
Highly metastatic
Validate
We test and validate the role of the discovered gene in driving metastatic disease.
We perform a series of extensive molecular and genetic studies to determine the role of the identified gene on driving metastatic disease in mice, utilizing state-of-the-art models including cell line and patient derived xenografts in 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.
Develop
Our experienced team develops new drug candidates suitable for clinical evaluation.
Having validated our targets through robust in vitro and in vivo phenotypic assays as well as genetically modified cells and models, we design and screen lead small-molecules or monoclonal antibodies that specifically alter the activity of the critical protein. Such leads are optimized to yield first in class therapeutics that exhibit strong activity against both primary tumor growth and metastatic progression in vivo.
SLC6A8 = Solute Carrier family 6 member 8
LXR = Liver X Receptor
Transform
We transform cancer medicine by leveraging RNA biology driven insights.
Inspirna’s deeply mechanistic studies that start with the identification of miRNA regulators in metastatic disease to the in vitro and in vivo validation of the downstream protein targets, coupled with our human clinicopathologic correlative data allow to design novel and innovative therapies. Our highly experienced clinical team uniquely positions Inspirna to advance these 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.