Akcea Therapeutics and Antisense Technology

Headquartered in Boston, Massachusetts, Akcea Therapeutics is a global biopharmaceutical company focused on development and commercialization of transformative treatments for serious and rare diseases. Akcea is an affiliate of Ionis Pharmaceuticals. By harnessing the power of proprietary antisense technology developed by Ionis, Akcea is advancing a mature pipeline of advanced RNA-targeted therapeutics to address significant unmet medical needs in 2 major disease areas: lipid-driven cardiometabolic disease and ATTR amyloidosis.

What are antisense therapies? Antisense therapies can be designed to inhibit the production of proteins involved in disease processes. Each antisense molecule targets a specific messenger RNA (mRNA) molecule, which normally carries the genetic instructions for making a particular protein from the DNA to the protein production complex in the cell. By binding to mRNA, antisense therapies prevent this process, thereby reducing the production of a disease-causing protein in a highly specific manner. Using this method of action, Akcea’s antisense therapeutics pipeline is designed to address diseases that other drug technologies, such as small molecules and antibodies have not been able to adequately address. For more information about Akcea, our portfolio, or Ionis’ antisense technology, please visit www.akceatx.com and www.ionispharma.com.