Lipid Nanoparticle Systems for Enabling Gene Therapies

Genetic drugs such as small interfering RNA (siRNA), mRNA, or plasmid DNA provide potential gene therapies to treat most diseases by silencing pathological genes, expressing therapeutic proteins, or through gene-editing applications. In order for genetic drugs to be used clinically, however, sophisticated delivery systems are required. Lipid nanoparticle (LNP) systems are currently the lead non-viral delivery systems for enabling the clinical potential of genetic drugs. Application will be made to the Food and Drug Administration (FDA) in 2017 for approval of an LNP siRNA drug to treat transthyretin-induced amyloidosis, presently an untreatable disease. Here, we first review research leading to the development…

• AP
  Alnylam Pharmaceuticals
• GB
  Genome British Columbia
• MR
  Medical Research Council Canada
• CI
  Canadian Institutes of Health Research
• NR
  National Research Council Canada