CRISPR meets LNPs: a new era in cell and gene therapies

Cell and gene therapies are entering a transformative era driven by the need for scalable, cost-effective, and safe gene editing and delivery technologies. While allogeneic cell therapies promise broader accessibility, traditional delivery methods—especially viral vectors—pose significant limitations, including high production costs, regulatory complexity, and safety risks. Electroporation, while non-viral, can negatively impact cell viability and consistency. Emerging as a powerful non-viral alternative, lipid nanoparticles (LNPs) enable efficient and scalable gene editing for both ex vivo T cell modification and in vivo gene therapy applications.

In this webinar, experts from Cytiva and Integrated DNA Technologies (IDT) present data demonstrating the potential of CRISPR-LNPs for multiplex gene editing in primary human T cells. Using a scalable NanoAssemblr™ microfluidics platform, LNPs were formulated with SpCas9 mRNA, Alt-R™ CRISPR-Cas9 sgRNAs, and a donor ssODN template. The results show high-efficiency editing, robust HDR, and high post-editing viability. Multi-tracking performance of CRISPR-Cas9 editing was also evaluated through the rhAmpSeq™ CRISPR Analysis System using next-generation sequencing.

The webinar also highlights the use of RNA-LNPs for in vivo gene editing, expanding their relevance beyond cell therapy.

Watch the webinar recording to explore how non-viral delivery platforms like LNPs could reshape the future of cell and gene therapy—from manufacturing and regulation to clinical translation.

Key learnings

In this webinar, we:

o Evaluate the efficiency of CRISPR-LNP-mediated multiplex gene editing in primary human T cells.
o Assess the scalability and viability of lipid nanoparticles as non-viral delivery platform for ex vivo and in vivo gene editing.
o Explore the non-viral delivery platforms like LNPs in the development and clinical translation of cell and gene therapies.
o Evaluate CRISPR off-target editing using rhAmpSeq-based next generation sequencing

Presenters:

Bernice Thommandru, Research Scientist III, Integrated DNA Technologies
Reka Geczy, Senior Scientist, Product Development, Cytiva

cytiva.com/AccelerateCTwithLNPs