• Challenges and considerations in early DP formulation development for NVGT
• Challenges in gene therapy LNP intravenous delivery compared to intramuscular vaccine delivery
• Modulating LNP tropism via passive delivery and active targeting
• Considerations in targeted LNP development

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.

Yuefei Shen

Associate Director CMC Drug Product Development

Sanofi