Silencing the messenger in elevated lipoprotein(a)

Elevated lipoprotein(a), or Lp(a), is a major risk factor for cardiovascular disease that affects one in five people worldwide, but currently lacks approved therapies. Here, Dr Giles Campion, EVP, Head of R&D and Chief Medical Officer of Silence Therapeutics, describes the company’s approach to developing an investigational siRNA therapy designed to reduce cardiovascular risk in people with elevated Lp(a).

Cardiovascular disease is the number one cause of death and disability worldwide.1 A major risk factor for developing cardiovascular disease is a condition called hyperlipidemia, in which a patient has abnormally high levels of cholesterol, fats and/or triglycerides in the blood. Most approved drug therapies for hyperlipidemia, including statins, work primarily by reducing levels of low-density lipoprotein cholesterol (LDL-C), which is often referred to as “bad cholesterol”. While these therapies can address the needs of patients whose hyperlipidemia involves elevated LDL-C, they cannot treat patients whose hyperlipidemia involves other factors, such as elevated levels of Lp(a).

Elevated Lp(a) is at the forefront of conditions for which small interfering RNA (siRNA) therapies are in development. siRNAs are targeted medicines with the potential to treat a wide array of diseases. Silence Therapeutics has multiple investigational siRNA therapies in development, including a clinical-stage siRNA designed to lower Lp(a) levels, which has the potential to reduce the risk of cardiovascular disease in patients with elevated Lp(a).

This article will cover siRNAs and their mechanism of action, the association of Lp(a) with cardiovascular disease, and the potential of an investigational siRNA therapy to address a major need among patients at risk of cardiovascular disease.