Antisense oligonucleotides (ASOs)
Akcea Therapeutics UK is focused on addressing the unmet needs and complex medical challenges of patients with important but less well-understood rare diseases. Our treatments are based on Ionis Pharmaceuticals’ proprietary antisense technology.
ASOs can be designed to inhibit the production of proteins involved in disease processes.1 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, ASOs prevent this process, thereby reducing the production of a disease-causing protein in a highly specific manner.2 Using this method of action, Akcea’s ASO pipeline is designed to address diseases that other drug technologies, such as small molecules and antibodies, have not been able to adequately address.
RNA is an optimal drug target for many reasons
The benefits of antisense drug technology
ASOs are designed to selectively target a specific gene-encoded mRNA molecule that carries the message for protein production.3
ASO discovery identifies the target responsible for a certain disease and then allows for development of a therapy with specific affinity to that target.3
Applying learnings from the testing of one chemically synthesised ASO drug to future ASO therapies increases the potential for success in early stages of development.3
Advances made in process chemistry have resulted in dramatic improvements in the simplicity of ASO drug production.3
References: 1. Visser ME, Witztum JL, Stroes ESG, Kastelein JJP. Antisense oligonucleotides for the treatment of dyslipidaemia. Eur Heart J. 2012;33(12):1451-1458. doi:10.1093/eurheartj/ehs084. 2. Phillips MI, Tang YL. Genetic modification of stem cells for transplantation. Adv Drug Deliv Rev. 2008;60(2):160-172. doi:10.1016/j.addr.2007.08.035. 3. Mansoor M, Melendez AJ. Advances in antisense oligonucleotide development for target identification, validation, and as novel therapeutics. Gene Regul Syst Bio. 2008;2:275-295. 4. Smith AP. Nucleic acids to amino acids: DNA specifies protein. Nature Education. 2008;1(1):126.