Small Molecule Target Biology & Validation
Through the integration of our sequencing-first assays and advanced bioinformatics workflows, we enable precise identification of disease-relevant and druggable RNA targets for small molecule discovery. Our platform reveals accessible regulatory regions, target engagement, and downstream biological effects that guide confident hit and lead selection. From early target discovery through off-target and mechanism-of-action validation, we provide the multidimensional data needed to de-risk small molecule development and accelerate progress toward the clinic.
Small Molecules
Effective small molecule therapeutics depend on selecting the right target and understanding how modulation impacts biology. Using sequencing-first technologies, we enable precise identification of disease-relevant RNA targets, validate mechanism of action, and detect transcriptome-wide off-target effects early. Our integrated approach provides a deeper view of target biology and therapeutic activity, helping teams make confident decisions and reduce downstream development risk.
Contact usTarget Identification
Mechanism-of-Action Validation
Off-Target Profiling
Find accessible regions to target
To be effective, therapeutics need to target unique and druggable regions of disease-causing genes. Learn how our solutions provide a comprehensive review of target biology allowing for optimal therapeutic design.
Identify open RNA structures
With our eSHAPE technology we can obtain validated information on the RNA secondary structure of a target gene. This structure information can be used to select drug candidates that target druggable pockets that are free of competition from proteins and miRNAs. It can also be used to directly measure where small molecules bind to a target RNA.
Predicted structures do not include the contribution of cellular factors that can limit accessibility. eSHAPE provides direct evaluation of structure in the cellular environment.
Locate unique druggable regions
With different diseases, a given gene can use different UTRs (regulatory regions outside the coding sequence). Our End-Seq technology directly identifies where UTRs are located, supporting the identification of druggable regions specific to the disease.
A 5’ UTR is more active in cancerous samples and not in healthy samples, making it a potential target for selective regulation in cancer.
Find protein effectors
RNA-binding proteins have been implicated in ASO activity and are frequent targets for small molecules and aptamers. With our RBP-eCLIP technology, we identify where a given protein binds across the transcriptome revealing information on potential proteins to block or mimic with small molecule drugs and to confirm protein activity for ASO efficacy.
Browser track showing the binding of a protein involved in splicing in the intron of RTEL1.
Validate
therapeutic effects
Many therapeutics have failed once they enter clinical trials due to limited on-target effects or problematic off-target effects. With our solutions we provide a holistic identification of on-target and off-target activity.
Transcription and translation effects
With eRibo Pro we directly measure changes in RNA expression (RNA-Seq) and translation (ribosome profiling), allowing for a comprehensive view of how a therapeutic affects cellular activity.
Identification of a stall in translation following treatment with the drug PF-846.
What our
partners say
"End-Seq assays are an invaluable part of our target assessment process at Ribometrix. The data are robust and often reveal novel 5’ and 3’ ends that aren’t reflected in public databases. Having this knowledge significantly influences strategic decisions around target selection in our RNA-targeting drug development process."
"Eclipsebio was instrumental in helping us characterize a small molecule-RNA interaction discovered by our platform. The Eclipsebio team worked closely with us to adapt their eSHAPE workflow to suit our needs, promptly delivered a complete data package, and generously followed-up."
De-risk small molecule discovery early
We deliver comprehensive insight into target biology, mechanism of action, and off-target liabilities, enabling smarter target selection and more efficient small molecule development.
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