Translational sciences


Turning today’s breakthroughs into tomorrow’s therapies. The Translational Science Group works within the Gene Editing Institute to bring innovative research from bench to bedside.

When bringing gene editing solutions to the clinic, there is a long road ahead for each therapy from discovery to implementation. Potential treatments must be both safe and effective. That work is the purview of our Translational Science Group. Using cutting-edge techniques and rigorously tested science, we drive research within the Institute and from the greater scientific community to bring these often-life-changing gene editing therapies to reality.

Translational Sciences


From concept to care. New approaches in cancer.

The Translational Science Group cooperates regularly with the FDA to bring gene editing therapies to the clinic. For example, we are currently advancing a gene editing-based treatment for non-small cell lung carcinoma that is now within the late-preclinical stage. This treatment, the first where CRISPR/Cas9 is used directly in solid tumors, is currently being prepared for submission as an investigational new drug.

The Institute’s Oncology efforts include:

  • Pioneering CRISPR tools to treat solid tumors
  • First in-human lung cancer applications
  • Reversing chemo-resistance
  • Treating inoperable, difficult-to-treat cancers
  • Viral and non-viral vectors
  • Precision delivery
  • Superior models for new research

Precision Genomics

New avenues. Innovative platforms.

The focus of our precision genomics group is to identify promising genomic targets to fuel innovative CRISPR techniques and technologies. By investigating potential drug targets, and how our current research can be expanded to encompass and treat new diseases, the Translational Science Group fast-tracks the discovery and development of new disease targets and therapeutic avenues.

Precision Genomics include:

  • Strong R&D pipeline of precision CRISPR therapeutics
  • Expanding approaches to solid tumors
  • Targeting squamous carcinomas, glioblastomas and inherited diseases

Genome Technologies

Novel approaches to broaden reach.

The Translational Science Group is pursuing innovative gene therapies and drug delivery mechanisms while expanding its suite of genome editing services and software. We generate new techniques and methods to enhance the CRISPR toolbox that gene editing scientists across the world can use to better lives and develop innovative new products.

We also offer labs worldwide the ability to jump-start their own research through the generation of custom gene edited cell lines.

Genome Technologies include:

  • SureKill improves CRISPR efficiency via tumor-specific mutation
  • CompleteKill treats broad chemoresistance
  • SlowKill and SelfKill increase CRISPR specificity
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