Exiqon’s antisense products are best in class in new comparative study

on November 5, 2015

Exiqon A/S (NASDAQ OMX: EXQ), a leading provider of flexible solutions for RNA analysis, today announced the impressive results of a comparative study on technologies for functional knockdown.

The study was conducted by Biogazelle, a Belgium company focusing on nucleic acid-based drug technologies for RNA-targeting therapies in cancer. Within this in vitro study, Exiqon’s Antisense LNA™ GapmeR technology was shown to be superior to other technologies tested for knockdown capability of lncRNAs (long non-coding RNAs).

Exiqon’s proprietary Antisense LNA™ GapmeR bioinformatics tools were used to design 200 LNA antisense molecules targeting 10 different targets selected by Biogazelle and tested for potency (knockdown efficiency) in two different cancer cell lines. The study demonstrated a remarkable overall success rate of 61%, which not only paves the way for Biogazelle’s therapeutics programs, but also brings yet another endorsement for Exiqon’s Antisense LNA™ GapmeR technology and bioinformatics tools. Exiqon’s products showed a success rate that was more than twice as high as the second best performing technology.

“We are extremely satisfied with the performance of Exiqon’s Antisense LNA™ GapmeRs for knockdown of lncRNAs” said CSO, Jo Vandesompele, Biogazelle, and continues; “With this set of encouraging results, we are very excited about the prospect of utilizing Exiqon’s platform for target validation and potential development of a new type of drugs targeting lncRNA”.

The Antisense LNA™ GapmeRs are high affinity antisense oligonucleotides used for functional analysis, allowing researches to study the gene function and downstream biological consequences of silencing a specific mRNA or lncRNA in cell cultures or animal models. The Antisense LNA™ GapmeRs may easily administered without transfection reagent.

Long-noncoding RNAs are a diverse group of RNA molecules that have attracted a lot of interest in the past few years as lncRNAs play a vital role in the homeostasis of cells. It is speculated whether drugs could be designed to target lncRNAs. However, the approach faces several challenges as lncRNAs not only forms inaccessible secondary structures but are also often retained in the nucleus and therefore hard to reach by competing knockdown technologies.

Exiqon offers a range of compatible LNA™ enhanced products for mRNA and lncRNA research including products for qPCR and in situ hybridization analysis. Information about the Antisense LNA™ GapmeRs is available here: http://www.exiqon.com/gapmers