Presenter:
- Dr. Samuel Clarke, Director of R&D, Cytiva, Canada
Delivering nucleic acids using lipid nanoparticles (LNP) to target cells has tremendous potential to enable new cell and gene therapies. In this presentation, we will discuss a genomic medicine toolkit composed of technologies that enable the development of RNA-LNP medicines. This includes off-the-shelf reagents for producing RNA-LNPs optimized for specific cell types, and the NxGen manufacturing platform designed for rapid development and scalable manufacture of RNA-LNPs from lab scale (mL) to GMP manufacturing (Liters).
We will showcase data in the areas of ex vivo CAR T cell and hematopoietic stem cell therapies where LNPs enable RNA delivery for transient expression of therapeutic genes and gene editing nucleases. In addition, we will share data demonstrating scale-up and manufacture of RNA-LNPs using a GMP-compliant process.
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