Targeted delivery to site of disease (e.g. atherosclerotic plaque) or therapeutic target (e.g. PCSK9 in the liver) |
Challenges and cost of scaled-up production |
Increased efficacy per drug dose |
Gap in knowledge of clinical safety |
Avoidance of systemic exposure and/or off-target effects |
Requirement for formulation-specific methods for characterization of composition and purity |
Potential for combining treatment, diagnosis, and monitoring disease in a single formulation |
Long-term stability of loaded agents |
Ability to be loaded with molecular recognition agents for “precision” therapy |
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