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Aptamer-Based Therapeutics Development

Development of Aptamer-Based Gene Therapies

To date, a variety of RNA aptamers have been developed as potential RNA therapeutics, offering unique clinical opportunities for the treatment of various cancers, cardiovascular diseases, and neurological disorders. Our company focuses on the continuing development of SELEX (Systematic Evolution of Ligands by Exponential Enrichment) technology, target identification, and new chemical approaches to provide our clients with specialized and extensive scientific services in the development of emerging aptamer-based therapeutics for rare diseases.

Background

Aptamers are oligonucleotides that have evolved in vitro or in nature and are attractive and viable alternatives to small molecule and antibody-based therapies. As a member of the emerging family of RNA therapeutics in the field of therapeutics, RNA aptamers are unique in that they act to disrupt at the protein level and not at the gene level. Aptamers are evolved by a method known as SELEX, resulting in structured oligonucleotide sequences with strict recognition and high affinity for the corresponding target molecules.

SELEX is a highly versatile technique that can be used to select aptamers with high affinity and specificity for a wide range of targets, including proteins, small molecules, and even whole cells. It has numerous applications in fields such as drug discovery, diagnostics, and biosensors, as aptamers can be used as a tool to target specific molecules or cells with high precision and specificity.

Aptamers have shown early promise as therapeutic agents. Several studies have demonstrated the therapeutic value of aptamer-based therapies in cancer treatment and in the treatment or identification of various neurological diseases such as multiple sclerosis (MS). Notably, aptamers also provide a novel strategy for delivering other therapeutic agents such as oligonucleotides and small molecules in a cell-specific manner. Advances in the use of combinatorial approaches that include aptamers as therapeutic molecules have opened up new avenues for the treatment of various rare diseases.

Fig. 1 The SELEX process.

Fig. 1 The SELEX process. (Zhou J, et al., 2012)

Our Services

  • Aptamer generation
    We provide our clients with a complete solution for aptamer generation, including library synthesis, aptamer selection, and biological function and characterization of the selected aptamers.
  • Custom aptamer service
    We help our clients synthesize custom high-affinity aptamers against targets of interest through base pair biotechnologies. Our service utilizes a unique form of multiplex SELEX technology to create RNA aptamers according to our client's needs.
  • Aptamer modification
    We improve the enzymatic resistance of RNA aptamers through chemical approaches such as the use of 2-modified nucleotides, locked nucleic acids (LNAs), and PEGylation. In addition, we can further enhance the biological potential of RNA adaptors by improving cellular delivery, duplex stability, and other properties as required by our clients.
  • Development of methods to regulate aptamer activity
    Antidotes are short complementary sequences (antisense) to the aptamers, which can reverse the inhibitory properties of the aptamers. We help customers develop derivatives of aptamer antidote pairs to regulate aptamer activity.
  • Development of functional aptamers against intracellular targets
    We help customers design aptamer expression cassettes with various promoters, including RNA Pol II (CMV, Hic, Mtn) and RNA Pol I and Pol III (tRNAU6, H1) to provide high intracellular levels of transcribed aptamers. In addition, we provide a "pure" aptamer in vivo expression system to ensure that the aptamers are transported and expressed within the cell in their correct folded and functional forms.
  • Development of RNA aptamer-mediated targeted therapy for rare diseases
    Aptamers exert therapeutic effects in a variety of human diseases by binding and blocking enzyme activity or protein-protein interactions. In addition, cell-specific aptamers have shown potential for therapeutic targeting or delivery. We help our clients develop aptamer-mediated targeted therapies to improve therapeutic indices and reduce the overall toxicity of drugs. We use a variety of strategies to functionalize RNA aptamers into cargoes or carriers.
    • Aptamer-mediated siRNA/shRNA delivery
      Development of non-covalent aptamer-siRNA conjugates
      Development of covalent aptamer-siRNA or shRNA chimeras
    • Aptamer-Mediated siRNA/shRNA-Loaded Nanocarrier Delivery
      Development of non-covalent aptamer-nanocarrier conjugates
      Development of covalent aptamer-nanoparticle conjugates

Our Aptamer-Based Gene Therapy Development Solutions

  • Development of RNA aptamers-based therapeutics for cancer, including target adherence factors, receptor tyrosine kinases (RTKs), immune system modulators, and modulators of cell growth.
  • Development of therapeutic RNA aptamers for the treatment of neurological diseases, such as MS and variant Creutzfeldt-Jakob disease (vCJD).
  • Development of RNA aptamers as anti-angiogenic agents.
  • Development of RNA aptamers as anti-coagulant agents.

With deep expertise and extensive research experience in the aptamer field, we are well-positioned to help our clients develop new RNA aptamer-based therapies for rare diseases. Our specialized scientific services can help you investigate the utility of aptamers as stand-alone therapeutics and establish multiple aptamer-mediated drug delivery systems such as aptamer-enzyme, aptamer-siRNA/shRNA, aptamer-nanoparticles, etc. If you are interested in our services, please contact us for more details.

Reference

  • Zhou, J.; et al. Current progress of RNA aptamer-based therapeutics. Frontiers in genetics, 2012, 3: 234.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

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