Target Identification and siRNA/ASO Screening

IntegrateRNA offers strong support in oligonucleotide drug discovery research from target validation, selection, and creation of oligonucleotide drug candidates to preclinical development. We have extensive experience in the discovery of mRNA-targeted drugs and in silico screening of ASO/siRNA. We use state-of-the-art technologies including bioinformatics, RNAi, high-throughput screening, along with profiling of target expression in relevant cell types which are available to help support your project.

Effectively identifying and validating therapeutic targets and biomarkers are key to effective precision therapeutic development. mRNAs currently the target of ASO and small interfering RNA (siRNA) drugs are increasingly being recognized as important drug targets that could eventually surpass the number of druggable proteins. Thus, the discovery of drugs that target mRNA is a promising direction for drug development and a new paradigm for the pharmaceutical industry.

Target Identification and Validation

Identify druggable mRNA targets associated with a disease of interest.
Confirm that mRNA targets are involved in disease etiology and result in a therapeutic benefit when altered.
Identify and optimize oligo molecules that have a pharmacological effect on the targets.

In Silico siRNA/ASO Screening

In silico oligonucleotide sequence design

Secondary structure prediction of the mRNA target.
Identification of preferable mRNA local secondary structures for ASO/siRNA binding.
Select the best predicted ASO/siRNA for docking and simulation.
Molecular dynamic simulation of the docked structure of mRNA-ASO/siRNA