Rare Diseases

Rare Diseases

Our commitment to rare diseases

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 technology

Our investigational treatments are based on Ionis’ proprietary antisense technology.

Antisense technology involves the use of synthetic nucleic acid sequences to interrupt the production of a specified protein by targeting the specific corresponding messenger RNA, or mRNA, that encodes that protein. In this way, antisense drugs can be used to reduce the level of proteins that cause, or contribute to, the progression of various diseases.

Our pipeline

Our current pipeline includes drugs with the potential to treat patients with disorders that other technologies, such as small molecules and antibodies, have not been able to adequately address.

While traditional drugs modify proteins involved in diseases, our treatments decrease the production of these proteins in the first place, offering the potential to significantly improve how serious and rare disorders are addressed.

Cardiometabolic lipid disorders

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