At Ionis, we've pioneered in RNA-targeted medicines for more than three decades, developing breakthrough medicines for people living with serious diseases – from rare conditions to some of the most prevalent health challenges. Our work is grounded in a deep understanding of how the body processes genetic information and a commitment to reaching people who have long had few or no effective treatment options for their conditions.
Despite the growing public conversation around RNA medicines, many people still have questions about what an RNA-targeted medicine is and how it works to address disease. Is it the same as gene therapy? Does it alter your DNA? Is it simply a variation of mRNA vaccines? In short – the answer is no.
RNA-targeted medicines are different
To understand what an RNA-targeted medicine is and what it does, it helps to first look at how the body functions. Your body is an intricate machine, and it all starts with your genetic code: DNA. Because it's so important, your DNA stays protected inside the nucleus of your cells, while RNA acts as a messenger to carry those instructions throughout the body. Those instructions guide the development of proteins, which perform many of the essential functions that keep the body working properly.
When RNA carries faulty or inaccurate instructions, important proteins might be missing or broken, or harmful proteins can be created. These disruptions can contribute to disease.
Today, many medicines work by managing those problematic proteins after the mistake has already been made. RNA-targeted medicines take a different approach. They act earlier in the process by interacting directly with specific RNA sequences to correct inaccurate instructions before problematic proteins can cause harm. Unlike gene therapy, which works by altering or replacing sections of a person’s DNA, RNA-targeted medicines do not alter a person’s DNA. Their effect occurs at the RNA level, working upstream of the problem, within the body’s natural systems. And unlike mRNA vaccines, which introduce new RNA instructions to trigger an immune response, RNA-targeted medicines target naturally occurring RNA to target the underlying cause of diseases.
This precision is what sets RNA-targeted medicines apart, and it is the foundation which Ionis has built its science for more than 30 years. For people living with serious diseases, many of which have lacked effective treatment options for decades, this approach represents a fundamentally different path forward.
Key Takeaways
- Your body is an intricate machine: DNA provides the code, RNA carries the instructions to build proteins that power vital functions.
- Targeting the underlying cause: Rather than managing broken proteins after they form, RNA-targeted medicines work earlier by interacting with specific RNA sequences to help alter protein production.
- Our foundation: Ionis has been a pioneer in RNA-targeted medicines for decades, with a growing portfolio of medicines for people living with serious diseases.