Aptamer Therapeutics

The specific and tailored target-binding properties of aptamers makes them ideal molecules for incorporation into a wide variety of Aptamer Diagnostic applications.

Many of these benefits also make aptamers excellent candidates for development of Aptamer Therapeutics.

  • Tailored specificity– aptamers can be selected to discriminate between closely related proteins, cells or tissue; limiting off-target effects.
  • Speed of development– fully automated discover accelerates hit-to-lead phase
  • Small Size– the small size of aptamers gives improved tissue penetration and uptake, and may be advantageous as more rapid clearance (of non-target-bound materials) may be beneficial in avoiding accumulation of toxic effects.
  • No immunogenicity– aptamers are normally non-immunogenic and do not require humanisation
  • Complex targeting – Aptamers can be selected to target important sites of interaction – disruption of protein-protein interactions, protein-nucleic acid interactions etc. Aptamers can be engineered to cross the blood brain barrier (BBB).
  • Excellent batch reproducibility – routine chemical synthesis approach to aptamer production gives greater control over batches, reducing variability; essential in drug development.
  • Simple formatting, modification and engineering– our optimised binders can be readily functionalised (during synthesis) and conjugated to a range of functional molecules.
  • Optimal Aptamer Drug Ratios (ApDRs)– Aptamer function and stability can be maintained when conjugating to drug molecules. Site specific labelling also eliminates the loss-of-function often observed with antibody labelling.
  • Multimeric affinity– aptamers can be multimerised to improve binding kinetics via avidity effects or to create dual function or bi-specific aptamers

These benefits have lead many research groups and Therapeutics companies, to isolate nucleic acids against disease associated proteins, to modulate their respective activities.

Aptamers are being increasingly employed in all phases of the drug discovery and development process; from biomarker discovery and target validation, to drug delivery or direct therapeutics and also as tools to assist in clinical trial e.g. companion diagnostics. Several aptamers have successfully entered clinical trials for different therapeutic indications and are extensively reviewed in the literature.

Aptamers can be selected against complex systems such as whole cells, viruses, bacteria, tissue sections etc. This direct selection approach is of considerable interest in the development of novel targeting agents, for the site-specific delivery of other ‘non-specific’ therapeutic cargos. Again, the specificity of these Aptamer-based delivery vehicles can be exploited to minimise off-target effects and allow repurposing of existing drugs by more specifically targeting them to the required site of action.

This aptamer-based approach offers a simpler alternative to drug delivery and bypasses many of the issues faced during the development of Antibody Drug Conjugates (ADCs). So called, Aptamer Drug Conjugates (ApDCs) are now a significant area of interest in a variety of applications, including targeted cancer therapies, novel targeted gene therapies etc.

Therapeutics Partnerships

We are looking to form collaborative partnerships within industry, government agencies and academic partners.

We have several projects underway which include on-going work with UK and international universities to provide access to aptamer technology on a fee-for-service or collaborative basis. We also work with leading scientists, medical specialists and technology companies in order to encourage growth, diversity and business opportunity through our supply chains and networks.

If you have an innovative idea for an aptamer use in the biotechnology sector then contact our innovation team to discuss funding/collaborative options.

References:

Theranostics 2018; 8(15):4016-4032. Aptamers in the Therapeutics and Diagnostics Pipelines Harleen Kaur, John G. Bruno, Amit Kumar, Tarun Kumar Sharma