Properties of Therapeutic Aptamers

This chapter contains sections titled: Introduction Aptamer Targets Cell Surface Targets Intracellular Targets Extracellular Targets Aptamer Binding Characteristics Aptamer Affinity Aptamer Specificity Aptamer Binding Kinetics Binding versus Function Chemical Modification of Aptamers 2′‐Modifications Capping the 3′ End Capping the 5′ End Phosphate Substitutions Base Modifications Polyethylene Glycol Lipid Tags Peptide Tags Routes of Administration of Aptamers Parenteral Administration Comparison to Biologics Opportunities for Alternative Aptamer Formulations Depot Topical Oral Pulmonary Ocular Aptamer Pharmacokinetics and Biodistribution Key Pharmacokinetic and Biodistribution Parameters Factors Governing Pharmacokinetics and Metabolic Stability of Aptamers Biodistribution of Aptamers Bioanalytical Methods for Aptamer Quantification Summary of Pharmacokinetic and Biodistribution Properties ofAptamers Toxicity Profile of Aptamers Immunogenicity of Aptamers Aptamer Manufacture Contributions to the Costs of Aptamer Synthesis Manufacturing Infrastructure Advantages of Chemical versus Biological Synthesis Examples of Therapeutic Aptamers in Development Antithrombin Aptamer ARC183 Anticomplement C5 Aptamer ARC187 Anti‐L‐Selectin Aptamer Anti‐PDGF‐BB Aptamer ARC127 Future Prospects for Aptamer Therapeutics References