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Target Validation and Therapeutics

Exiqon Custom Pharma Services design and supply third generation LNA™-enhanced antisense oligonucleotides for potent gene silencing of any RNA target, or modulation of microRNA activity. LNA™ antisense oligonucleotides are ideal for both in vitro and in vivo use, and show great promise as therapeutics of the future.

Exiqon, now a QIAGEN company


Exiqon and QIAGEN have joined forces to bring you an enriched product and service offering that builds on the expertise and product synergies of the two companies.

Effective 1 October 2017, Exiqon products and services are fully integrated with QIAGEN, and available for purchase only through QIAGEN.

Meet the new face of Exiqon Services.
  • Potent gene silencing of any mRNA or lncRNA using Antisense LNA™ GapmeRs
  • Reach undruggable targets through modulation of microRNA function
  • LNA™ microRNA Inhibitors, Mimics and Target Site Blockers
  • Use in vitro and in vivo for target validation or therapeutics
  • Custom design tailored to your requirements by our LNA™ experts
  • Expert consultation on in vivo projects

Potent knockdown in vitro and in vivo

Antisense LNA™ GapmeRs enable potent and specific knockdown of messenger RNA or long non-coding RNA (lncRNA) targets. Antisense LNA™ GapmeRs are active in vivo (with no formulation), making them an excellent alternative to siRNA.

"We are extremely satisfied with the performance of Exiqon's Antisense LNA™ GapmeRs for knockdown of lncRNAs. We are very excited about the prospect of utilizing Exiqon's platform for target validation".
Prof. Vandesompele, CSO Biogazelle

Unrivalled performance in vivo

Excellent pharmacokinetic and pharmacodynamic properties of Antisense LNA™ GapmeRs have been demonstrated in many different tissues and organs. LNA™ antisense oligonucleotides are well tolerated and show low toxicity in vivo. Effective and long lasting knockdown of mRNA and lncRNA can be achieved in a broad range of tissues with Antisense LNA™ GapmeRs administered in live animal models (Figure 1).

"Antisense LNA™ GapmeRs target the lncRNA directly and allowed us to test the therapeutic potential of Chast silencing in animal models."
Prof. Thomas Thum, Hannover Medical School (Read interview)

Therapeutic applications

Several pharmaceutical companies including Roche are driving development of antisense therapeutics, signaling that antisense technology is becoming the third major modality in the drug development industry. Over 76 antisense oligonucleotides have been tested in human clinical trials (reviewed by Sharma et al., Antisense oligonucleotides: modifications and clinical trials. Med. Chem. Commun. 2014, 5, 1454-1471. DOI: 10.1039/C4MD00184B). In 2013, the antisense therapy mipomersen (marketed as Kynamro) was approved by the FDA.

"Tumor growth was significantly inhibited in mice injected with the SAMMSON-targeting Antisense LNA™ GapmeRs."
Prof. Jean-Christophe Marine, K.U. Leuven (Read interview)

Design tailored to your requirements

Through Exiqon Custom Pharma Services, you have access to Exiqon's bioinformaticians who are pioneering the design of Antisense LNA™ GapmeRs, enabling the design process to be tailored to your specific requirements.

Expert consultation on in vivo projects

Exiqon Custom Pharma Services have experience developing Antisense LNA™ GapmeRs for therapeutic use, and can assist with selection of super-potent Antisense LNA™ GapmeRs to ensure maximal silencing of the RNA target in vivo, as well as advise how to monitor in vivo biodistribution. We can suggest the best approach when using Antisense LNA™ GapmeRs for target validation, including administration in vivo animal models.

Antisense LNA™ GapmeRs are available from Exiqon in a range of purities and quantities – right up to high quality syntheses at mg – kg scales for in vivo use.

Figure 1 TEXT
In vivo knockdown using Antisense LNA™ GapmeRs. (Click to learn more)

Reach undruggable targets

MicroRNAs themselves are potential targets for therapeutic drugs. MicroRNAs interact with multiple target genes, therefore modulators of microRNA activity represent a new class of drugs that have the possibility to alter whole networks of gene expression, and reach previously undruggable targets.

MicroRNA activity can be modulated through the use of microRNA mimics (also known as microRNA replacement therapy) or microRNA inhibitors (also known as anti-miRs or antagomiRs). MicroRNA therapeutics using both approaches are currently in development (Figure 1).

Proven LNA™ technology for microRNA Therapeutics

Exiqon's LNA™ technology is widely used in the synthesis of microRNA inhibiting drugs. Effective microRNA inhibition has been achieved in multiple organs and tissues by systemic and local administration of custom designed In vivo LNA™ microRNA Inhibitors (Figure 2). Successful phase 2 human trials with a LNA™ miR-122 inhibitor for treatment HCV infections are a testimony to the unique drug like properties of these short antisense molecules (Janssen et al. Treatment of HCV infection by targeting microRNA. N Engl J Med. (2013) 368, 1685-94).

For the precise disruption of the regulation of one specific RNA target by masking a microRNA binding site within it, Exiqon have developed LNA™ microRNA Target Site Blockers. More recently, Exiqon has developed sophisticated third generation LNA™-enhanced microRNA Mimics.

Design tailored to your requirements

Through Exiqon Custom Pharma Services, you have access to Exiqon's bioinformaticians who are pioneering the design of LNA™ microRNA therapeutics including:
  • microRNA mimics
  • microRNA inhibitors
  • microRNA target site blockers
Our bioinformaticians can tailor the design to your specific requirements.

Expert consultation on in vivo projects

Exiqon Custom Pharma Services can advise you on their administration in vivo animal models. LNA™ microRNA mimics, inhibitors, and target site blockers are available from Exiqon in a range of purities and quantities. Inhibitors and target site blockers are available right up to high quality syntheses at mg – kg scales for in vivo use.
Figure 1 MicroRNA therapeutics in development
In vivo knockdown using Antisense LNA™ GapmeRs. (Click to learn more)

Figure 2 Successful inhibition of microRNAs in a variety of tissues
Successful inhibition of microRNAs in a variety of tissues. (Click to learn more)

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