Turning today’s breakthroughs into tomorrow’s therapies. The Gene Editing Institute utilizes cutting-edge CRISPR-directed gene editing 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. Our research utilizes a cell-free gene editing system developed in-house as a platform to investigate unique and often untreated genetic diseases. This unprecedented system allows researchers to control and interrogate the process of CRISPR gene editing like never before. By removing the gene editing reaction from the nucleus of the cell and allowing direct interrogation of every step of CRISPR-mediated DNA cleavage and its subsequent DNA repair mechanisms, the cell-free gene editing system provided never-before-seen clarity into gene editing reactions that can dramatically accelerate research in specific genetic diseases.

Potential treatments must be both safe and effective. Using novel 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. We also expand the scientific field’s knowledge and understanding of DNA repair, and how different CRISPR targets can be designed to effectively target genes to alleviate diseases.

The Gene Editing Institute's Translational Research includes:
  • Leading expertise in a collaborative research community
  • Robust pipeline of novel CRISPR modules
  • Development of tools to mitigate the failure of standard oncology and inherited disease care
  • Expansion of CRISPR research into novel technologies to broaden reach


From concept to care. New approaches in cancer.

The Gene Editing Institute cooperates regularly with the FDA to bring gene editing therapies to the clinic. For example, we are currently advancing gene editing-based treatments for non-small cell lung carcinoma, head and neck cancer, and glioblastoma now at 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.

  • 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

New avenues. Innovative platforms.

The focus of our precision genomics research 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, we fast-track the discovery and development of new disease targets and therapeutic avenues.

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

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