At EmendoBio, we do not believe in a one solution fits all gene editing approach. We believe the pathway to curing diseases is a tailor-made custom solution per target.

The foundation of our discovery platform is a continuously evolving panel of novel nucleases, each with different characteristics and applications, that allows us to dramatically broaden the area of the genome that is targetable.

To achieve the optimal gene editing solution for each disease, we use AI based computational prediction approach and protein engineering to create the best nuclease available for each specific indication, optimize the gene editing composition for that target, and develop a custom drug composition with ultra-high target specificity and elimination of off-target effects.

 

Discover

We start by analyzing the disease characteristics, mechanism of action, and genetic alterations, then design an editing strategy that would address as much of the patient population as possible.

  • Genetic analysis (Pseudogenes, copy number)
  • Required editing (KI, KO)
  • Design specific editing strategy
  • Select and test guides
  • Discover and test nucleases
  • Verify editing on genomic, mRNA and protein levels
  • Verify functional output in model cells
  • Verify functional output in patient-derived cells
  • Determine off-targets, inversions and translocations

Optimize

The most effective strategy moves into Lead Optimization where it is optimized through protein engineering, guide design and library selection.

  • Optimize guide per target
  • Optimize nuclease per target
  • Protein engineering
  • Libraries selections for variants
  • Monitoring on and off-target
  • Finalize composition

License out

We offer our panel of OMNI nucleases and off-the-shelf compositions for out-licensing.

  • Multiple OMNI nucleases with desired features
  • Already developed editing solutions
  • Development of novel editing solutions
  • Consultation on tailoring gene editing solutions for your target
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