With RNA as the target that forms the basis of our novel drug discovery platform, we are tackling an unprecedented range of diseases across a broad set of therapeutic areas. Our antisense therapies are disrupting diseases and, we hope, changing their course—from the rarest of conditions, to those that impact millions of people.

And as we continue to advance our technology, our therapies become more precise and more potent, creating opportunities for us to efficiently bring transformative medicines to patients.

Antisense: changing the course of disease

To appreciate how Ionis is using antisense technology to revolutionize drug discovery, we must first look at how traditional medicines work in the body.

Traditional approaches vs. antisense. The human body is made up of billions of cells, each containing the unique genetic information, or DNA, that defines a person. Contained within DNA are instructions for making all the proteins in the human body.

Proteins are crucial building blocks for the structure and function of all organs and systems within the human body. But over or under production of a protein, or production of a mutated protein, are common causes of many human diseases. For more than 100 years, most traditional medicines like small molecule inhibitors or, more recently, antibody-based therapies have worked to target these proteins once they are produced and doing damage in the body.

Antisense therapies change the process of producing a protein before it even begins. To build a protein, a cell must make a copy of the DNA, which contains specific instructions for how to make that particular protein. This copy, called messenger RNA (mRNA), carries the instructions to the part of the cell where proteins are made.

Antisense therapies are designed to seek out, bind to and destroy a mRNA in a highly specific manner, so that the amount of disease-causing protein is dramatically decreased. Antisense therapies can also treat diseases caused by too little protein by increasing the production of the protein, thereby restoring the protein to normal levels.