Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria

• Published in: Clinical pharmacokinetics Vol. 60; no. 3; pp. 365 - 378
• Main Authors: Badri, Prajakta, Jiang, Xuemin, Borodovsky, Anna, Najafian, Nader, Kim, Jae, Clausen, Valerie A., Goel, Varun, Habtemariam, Bahru, Robbie, Gabriel J.
• Format: Journal Article
• Language: English
• Published: Cham Springer International Publishing 01.03.2021; Springer Nature B.V
• Subjects: Antibodies; Complement C5 - pharmacokinetics; Gene expression; Healthy Volunteers; Hemoglobinuria, Paroxysmal - drug therapy; Humans; Internal Medicine; Liver; Medicine; Medicine & Public Health; Monoclonal antibodies; NCT; NCT02352493; Original; Original Research Article; Pharmacodynamics; Pharmacokinetics; Pharmacology/Toxicology; Pharmacotherapy; Proteins; RNA Interference; RNAi Therapeutics

Background Cemdisiran, an N -acetylgalactosamine (GalNAc) conjugated RNA interference (RNAi) therapeutic, is currently under development for the treatment of complement-mediated diseases by suppressing liver production of complement 5 (C5) protein. This study was designed to evaluate the safety, tol...