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Ionis Innovation

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.

Antisense Technology

Our antisense therapies, also known as antisense oligonucleotides, or ASOs, are designed to bind precisely with RNA, halting the process of creating a disease-causing protein.

Healthy person

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Person with disease

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Person treated
on antisense drug

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The Ionis antisense advantage

Optimizing antisense through advancements in medicinal chemistry and pioneering new ways to deliver antisense has been a central focus of our research efforts, allowing us to bring options to those who have had none.

A perpetual and efficient innovation machine

Our platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed to treat a broad range of diseases.