In recent years, significant discoveries, some unprecedented, have been made in molecular and cell biology, which offer improved opportunities for advanced therapies development.
We at Selecta Biotech believe, that eliminating drug promiscuity and inherent toxicity is the key to success in development of highly effective and, most importantly, safe therapeutics that have the potential to redefine treatment outcomes in a broad range of currently intractable and devastating diseases affecting either smaller or larger patient groups.
The development of new drugs requires two major steps: the identification of a therapeutically relevant target and the development of a compound capable of modulating its function. An increasing understanding of the scientific underpinnings of the biological causality of many diseases have led to an upsurge in the number of potentially attractive molecular targets for therapy.
Each of us carry a unique genetic information, or DNA, that defines all the proteins in the body. Proteins do most of the work in our cells and are required for the structure, proper function, and regulation of the body´s tissues and organs.
To build a protein, a cell must make a copy of DNA, named messenger RNA (mRNA), which carries specific instructions for how to make that particular protein. If the instructions in the mRNA are defective, a pathologic protein is made and may cause a human disease. Traditional therapeutic approaches have been focused on inhibition of these defective proteins.
Unlike conventional therapeutics, oligonucleotides have a distinguishing feature in that they can halt the process of producing a pathologic protein before it even begins. They recognize and bind to mRNAs, thereby either initiating their destruction or blocking them from further processing. By doing so, pathologic proteins are not even built and cannot do damage in the body.
Oligonucleotide therapeutics have generated high expectations stemming from their potency and have enjoyed remarkable success in clinic within the past years. Irrespective of the promising results though, there is a long-lasting stagnation in overcoming drug promiscuity responsible for many clinically relevant side effects or adverse events.
Our therapeutic oligonucleotides based on the patented ESiNAR-X® platform
represent promising tools to meet this challenge.