Optimer® delivery vehicles enable targeted delivery of diverse payloads to challenging cell types
The advent of oligonucleotide therapeutics has given rise to novel drug development strategies. Oligonucleotide ligands can operate as both independent therapeutic modalities and as delivery vehicles for diverse payloads with the potential to enhance the effectiveness of current medicines. Optimer technology is an example of oligonucleotide-based ligands that can be used to modify intracellular disease targets and for the targeted delivery of payloads. This work demonstrates the power of Optimer technology as a therapeutic tool able to directly modify gene splicing in chronic myelomonocytic leukaemia (CML) and as delivery vehicle targeting multiple myeloma cell phenotypes. Optimer ligands can be successfully isolated for specific cell targeting and internalisation, showing no off-target interactions, and allow simple site-specific conjugation of desired payloads. This flexibility of Optimer ligands to act as direct therapeutics or enhance the therapeutic index of current gene therapy and small molecule drugs as delivery vehicles could improve patient tolerability and safety profiles while progressing development pipelines for the pharmaceutical industry.