The discovery of Cas9 revolutionized how genetic disease can be treated, creating new and exciting opportunities. The ability to cut DNA and change, insert or remove elements of targeted genes was game-changing, putting potential cures for patients with genetic disease within reach. While patients may soon reap the benefits from first-generation CRISPR-based medicines, the field continues to expand, unearthing a next-generation of genomic therapies that hold the potential for more precise gene editing to treat many more diseases and many more patients.

With a suite of nucleases with the PAM diversity to target more than 93% of the human genome, our toolbox enables a variety of functions, spanning from simple knockdowns of a protein to more sophisticated and precise ways of editing the genome. We have chosen to focus our efforts on four primary platforms for editing DNA, each of which contain unique properties that enable a differentiated approach that can be used for many diseases.

Discovery Approach

We take a multi-step approach to drug development, beginning with our proprietary discovery engine which is powered to discover novel editing approaches that enable new therapeutic programs with differentiated editing capabilities.

Source From Nature

Employ sophisticated methods of curating metagenomic data

Source From Nature

3 billion+ proteins indexed to date

AI/ML Optimization

High-throughput screening to identify and optimize editing technologies

AI/ML Optimization

AI/ML feedback loops used throughout discovery and optimization to improve speed/efficiency


New discoveries with novel properties


We have discovered 9 proprietary gene editors and 6x more nuclease families than are published in the literature


Optimized engineering to significantly improve editing efficiency

Engineering has generated >30x improvement in editing efficiency from the wild type nuclease to achieve therapeutically relevant levels of editing efficiency

Building a

Toolbox Boxes

Technology Platform

Our Differentiation

Bespoke Nucleases

Knockdown +

  • Nucleases with large deletion patterns
  • Minimal guide RNA confers manufacturing advantages
  • Small Cas effectors easily fit into AAV
  • Cas12 more specific than Cas9
Precision Editing

Precision (RT) Editing

  • Precision correction without bystander effect or windowing limitations
  • PAM diversity

Nuclease Excision

  • Small size of effectors enables AAV delivery
  • Can use single or double guides depending on size
CRISPR Transposases

Large Insertions

  • Enables more natural gene regulation
  • Treatment is more durable versus AAV
  • Single treatment to treat multiple patients with different mutations


Tackling genetic disease is complicated, so, we begin with the important questions: What are we trying to fix? Having many genomic editing tools provides Arbor with the unique ability to work backward from disease pathology and choose the optimal editors or combination of editors for the disease in question.

Arbor® Publications

. Nature Communications; 20 May 2022; Vol 13 (2833)
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. Nature Reviews Microbiology; 2020; Vol 18: pp67-83
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. Nature Reviews Microbiology; 05 Jun 2019; Vol 17: pp513-525
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. Science; 6 Dec 2018; Vol 363 6422: pp88-91
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. Molecular Cell; 19 Apr 2018; Vol 70 (2) 99: pp327-339.E5
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