Advances in molecular and cell biology have transformed our understanding of disease and opened new possibilities for developing targeted therapies. Our focus is on what comes next – reducing off target effects and unwanted toxicity while improving efficacy to develop treatments with stronger safety profile and greater clinical benefit.
The development of new drugs requires two major steps: identifying a molecular target that plays a key role in the disease, and designing a therapeutic capable of modulating its function.
Our genetic information is stored in DNA. Before a protein is made, the cell creates a temporary copy of the genetic instructions called messenger RNA. If those instructions are faulty, the cell can produce a protein that contributes to disease. Most conventional medicines act after the protein has already been made.
Oligonucleotides work one step earlier. They recognise and bind to specific RNA molecules – initiating their destruction or blocking further processing. As a result, disease-causing proteins are prevented from being produced in the first place.
Oligonucleotide therapeutics have shown remarkable clinical success and continue to expand the possibilities for treating disease. Still, there is room to improve. Unwanted interactions beyond the intended target can reduce therapeutic performance and contribute to clinically relevant side effects. Our patented ESiNAR-X® platform was designed to overcome this limitation and help unlock more of the potential of oligonucleotide therapeutics.
Our proprietary ESiNAR-X® platform is built around an unconventional structural design aimed at improving the properties of therapeutic oligonucleotides. Through careful molecular engineering, we aim to enhance therapeutic performance, improve specificity, and address key limitations of current approaches.
Designed to selectively recognize target RNA while minimizing interactions with closely related sequences.
Supports efficient internalization into cells, with the potential to enable less frequent dosing and lower administered amounts.
Preclinical studies showed excellent tolerability, supporting the potential for and improved safety profile.
Triggers biologic effects only in cells containing the intended target RNA, supporting precise therapeutic action.
Enables efficient reduction of target RNA levels to as low as 0%, providing robust modulation of disease-relevant pathways.
Can be adapted to a wide range of RNA targets, expanding opportunities across different therapeutic areas.
A key feature of ESiNAR-X® is its ability to enter cells without relying on additional delivery systems. Demonstrated across multiple cell types, including stem cells, and supported by favorable biodistribution profiles in vivo, this property may enable more efficient dosing strategies while maintaining therapeutic performance and supporting improved safety.
Drag to compare — spontaneous internalization of ESiNAR-X® (green) in selected cell types.
See how ESiNAR-X® translates into therapeutic leads across oncology, infectious and renal diseases.
