Small Interfering RNA (siRNA) Therapy

Excerpt

This activity focuses on small interfering RNA (siRNA) therapy, exemplified by FDA-approved agents such as patisiran, givosiran, lumasiran, inclisiran, nedosiran, and vutrisiran. These therapies are pivotal in managing rare metabolic ailments, including hereditary transthyretin amyloidosis (hATTR), acute hepatic porphyria (AHP), primary hyperoxaluria type 1 (PH1), and lowering low-density lipoprotein cholesterol (LDL-C) in subjects with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD). The activity will discuss the mechanism of action of siRNA, its adverse event profile, and other key factors essential for interprofessional team members involved in patient care. Furthermore, participants will gain insights into administration techniques, contraindications, clinical toxicology, and monitoring strategies necessary to safely and effectively utilize siRNA therapies in clinical practice.