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Webinar

Towards CRISPR-on-Demand: A Platform Manufacturing Approach for Gene-edited Cell Therapies

ET
60 Minutes

For patients with rare genetic diseases, the window to treat is often short. Dr. Ke Li, Scientific Director of the Investigational Cellular Therapy Program at the University of California, San Francisco (UCSF), will share how his team is building a CRISPR-on-Demand platform to bring personalized gene-edited cell therapies to the clinic faster, using primary HLH as a model. He will also cover a non-viral genome engineering approach to ultra-large DNA knock-in for next-generation cell therapies. 

Dr. Garrett Rettig (IDT) will follow with data on choosing the right gRNA grade at each stage of development. The collaborative comparability study he will present shows gRNA purity drives on-target potency, and off-target profiles from screening-grade gRNA closely match engineering-grade material, so early safety data carries forward.

What you will learn:

  • A platform framework for developing N=1 gene-edited cell therapies
  • How Quality-by-Design guides clinical-scale, non-viral T cell manufacturing
  • How knock-in correction restored perforin expression and T cell function
  • Extending non-viral genome engineering to ultra-large DNA knock-in for next-generation cell therapies
  • How gRNA grade affects purity, editing, and off-target analysis

Speakers

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Ke Li, Ph.D.

Scientific Director, Investigational Cellular Therapy Program
University of California, San Francisco (USCF)

Ke Li is the Scientific Director of the Investigational Cellular Therapy Program at the University of California, San Francisco (UCSF), where he leads process and analytical development efforts for ex vivo cellular therapies targeting hematologic malignancies, solid tumors, and rare diseases. His work focuses on optimizing non-viral genome and epigenome engineering platforms and has been instrumental in the development and clinical translation of UCSF’s first CRISPR-engineered CAR T therapy towards clinical manufacturing. Dr. Li brings extensive experience in CMC strategy, IND-enabling studies, regulatory affairs, and technology transfer, and has led multiple collaborations spanning academia and industry. Dr. Li earned his Ph.D. in Molecular Genetics from Albert Einstein College of Medicine and completed postdoctoral training at the Howard Hughes Medical Institute (HHMI)/UCSF.

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Garrett Rettig, PhD

Principal Scientist, Genome Editing Analysis
Integrated DNA Technologies (IDT)

Garrett Rettig leads the Genome Editing Analysis team in Research and Development. Garrett is a Principal Scientist and has been at IDT for 18 years. The team is focused on continued innovation in assay development related to the safety of CRISPR editing. Specifically, they are developing NGS-based assays to measure the efficiency and specificity of established and gene editing systems.

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Rolf Turk, PhD

Senior Director of Innovation Programs
Integrated DNA Technologies (IDT)

Rolf Turk, PhD is Senior Director of Innovation Programs at Integrated DNA Technologies (IDT), where he leads strategic collaborations and external innovation initiatives focused on genomic medicine, genome editing, and advanced molecular technologies. He serves as Alliance Manager for the Danaher–IGI Beacon for CRISPR Cures program and directs partnerships spanning academia, biotechnology, and industry to accelerate translation of emerging scientific discoveries into impactful solutions. Prior to his current role, Dr. Turk held scientific leadership positions in genomic medicine and genome editing at IDT and conducted genetic research at the University of Iowa. He holds a PhD in Human and Clinical Genetics from Leiden University. His work combines deep scientific expertise with a passion for building collaborative ecosystems that enable innovation in cell and gene therapy, genome editing, and precision medicine. 

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