Transthyretin amyloidosis is caused by the deposition of transthyretin amyloid in peripheral nerves and the heart. RNA interference (RNAi) targeting messenger RNA transcribed from a disease-causing gene could theoretically be used in the management of the disease by reducing the production of transthyretin. Coelho et al. identified a potent antitransthyretin small interfering RNA, which was encapsulated in two distinct formulations of lipid nanoparticles, ALN-TTR01 and ALN-TTR02. Each formulation was studied in a single-dose, placebo-controlled phase 1 trial to assess safety and effect on transthyretin levels. Both formulations suppressed the production of transthyretin (both mutant and nonmutant forms) in this proof of concept study.
Safety and efficacy of RNAi therapy for transthyretin amyloidosis. Coelho et al. N Engl J Med. 2013 Aug 29;369(9):819-29. PMID: 23984729
Posted by Yannis Trakadis, MD