Lipid nanoparticle (LNP) technology, as currently one of the most promising and emerging technologies, shows the ability to deliver nucleic acid therapeutics for non-viral gene therapy (NVGT). Compared to intramuscular vaccine delivery, intravenous (i.v.) delivery of a LNP formulation for gene therapy shows unique challenges. A LNP formulation for gene therapy via i.v. may require different formulation and process design. Here, we will discuss the considerations and challenges in early LNP development for NVGT.
Learning Objectives:
What are the considerations in early DP formulation development for NVGT
Compared to the intramuscular mRNA vaccine, what are the challenges and how to overcome the challenges in intravenous NVGT gene therapy
What are the considerations in standard and targeted LNP development
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