Advancing RNA research and therapies with Optimer

From the RNA Leaders US Congress last week to Oligonucleotide Therapeutics and Delivery this week, our team are busy learning, networking and discussing on both sides of the pond about the latest advances in RNA therapies. With this in mind, we are taking a quick look at some of the ways Optimer technology supports the field.

Optimer binders are oligonucleotide-based antibody alternatives that can support RNA research from novel assay reagents to targeted therapies. As oligonucleotides, they can be RNA-based or DNA-based according to the needs of the assay or therapeutic. These high affinity and specificity binders are offering new solutions to the field of RNA research.

Targeted delivery of RNA therapies

Optimer binders can be easily conjugated to RNA therapies to act as delivery vehicles for siRNA, saRNA, ASO, and larger nanoparticles to guide the delivery of mRNA. The controlled chemistry of Optimer binders allows for known drug-Optimer ratios, and with the ability to undergo receptor-mediated endocytosis and access cells through pinocytosis, there is high potential for improving intracellular delivery of functional payloads with Optimer delivery vehicles. What’s more, the half-life of Optimer reagents can be tuned to allow for rapid hit-and-run strategies or longer half-lives as needed.

Site-specific delivery remains a significant challenge faced by novel RNA therapies. The use of cell or tissue targeting Optimer non-viral vectors can help to overcome the three major hurdles of:?

1. selective targeting to specific cell types,?
2. internalization of the RNA therapeutic payload to the target cell, and?
3. ensuring the therapy is functional?once in the cell.?

To find out how we develop Optimer as therapeutic vectors that overcome each of these challenges to advance your precision therapies, join our webinar this week or register to receive the post-event recording.

Optimer therapeutics

Beyond delivering RNA therapies to a cell, Optimer binders can act directly as a therapeutic. Optimer binders that inhibit enzymes or prevent specific protein-protein interactions can act as novel therapeutics within themselves. Working with scientists at Cancer Research UK, we developed an Optimer to inhibit an enzyme in cancer, with the result of reducing aberrant splicing of specific transcripts.

To find out more about the different potential strategies for Optimer binders download our application note.

Gene switches

RNA Optimer binders are being explored as gene switches. Optimer binders can bind small molecules with high selectivity and affinity. When combined with different expression platforms the Optimer can act as a switch to control gene expression in diverse contexts. Target binding leads to a conformational change that causes structural changes that can allow expression or sequester the expression platform by acting as a riboswitch.

Assay reagents

Optimer binders to specific targets can be used as assay reagents for research, bioanalytical and QC assays. The in vitro development methods of Optimer binders overcome the reliance on the immune system that antibodies have, meaning there is a wider target range for these binders to support novel targets. We have worked with partners to develop Optimer binders to act as PK reagents, bioanalytical reagents for patient stratification, research reagents for transcriptomic targets, vaccine adjuvants and reagents that support the manufacture of mRNA vaccines.

We partner with scientists from the brightest minds in academia to leaders of the pharma sector to support RNA research and we are loving engaging with the community to learn more about the latest developments.