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The discovery of CRISPR/Cas9 as a simple and effective tool for targeted modification of cells has opened up new possibilities for gene or cell therapies. Join us to hear about strategies for using CRISPR/Cas9 for curing genetic diseases, e.g. sickle cell disease or HIV, its opportunities and challenges. In addition, we will discuss how CRISPR/Cas9 can impact next-generation disease model systems and drug screening, which might be relevant for combinational therapies.

Who Should Attend

  • Researchers working on gene or cell therapies
  • Translational research scientists
  • Clinical researchers
  • Process development scientists and pilot-scale scientists
  • Chief scientific officers and chief technical officers
  • CMO scientists

You Will Learn About

  • Introduction to CRISPR/Cas9
  • Application examples with focus on use for sickle cell disease
  • Opportunities and challenges for the use of CRISPR/Cas9 in gene/cell therapeutic applications
  • Use of CRISPR for next-generation disease model systems
  • Non-viral transfer of CRISPR/Cas9 cargos and up-scaling of the transfection process

Produced with support from:

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Matt Porteus, M.D., Ph.D.
Associate Professor of Pediatrics,
Stanford University

Gregory Alberts, Ph.D.
Global Subject Matter Expert,
Lonza Bioscience Solutions