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Nanomedicine and Nanoscale Delivery (Focus Group – NND)

CRISPR/Cas9 Nanoparticle Formulations: From In Silico Insights to Microfluidics and Stability Strategies

Patrícia Henriques – Scientist, Hovione; Susana Ramos – Senior Analytical Scientist, Hovione; Paulo Lino – Senior Scientist, Hovione; Diogo Salgueiro – Associate Scientist, Hovione; Vitor Francisco – Principal Investigator, CoLAB4Ageing; Artur Filipe Rodrigues – Postdoctoral Research Associate, CNC - Center for Neuroscience and Cell Biology; Susana Simões – Researcher, CNC - Center for Neuroscience and Cell Biology; Lino Ferreira – Investigator Coordinator, CNC - Center for Neuroscience and Cell Biology; Eunice Costa – Fellow scientist, Hovione
Introduction:

Neurological disorders, including stroke and neurodegenerative diseases, cause severe disability and lack treatments to replace lost neurons1. The REGENERAR project aims to develop non-viral, engineered nanoparticles (NPs) to epigenetically reprogram non-neuronal cells for brain regeneration2–4. This work focuses on transitioning CRISPR/Cas9 polymeric NP formulations to pharmaceutical standards, focusing on colloidal stability, size control, and reproducibility. Cas9 was used as a model for dCas9, the nuclease inactive (“dead”) Cas9 variant that will be employed in later stages. Strategies included in silico screening, microfluidics manufacturing, and steric stabilization.

Learning Objectives:

  • Understand the role of formulation and process parameters in nanoparticle quality.
  • Evaluate strategies to improve colloidal stability of CRISPR/Cas9 nanoparticles.