At Arbor, we envision a world free of disease by using genetic medicines to fundamentally shift the paradigm from treatment to functional cure, and ultimately to prevention. We aim to accomplish this through the discovery of programmable DNA editors with the power to enable bespoke curative genetic medicines for patients.

We believe gene editing therapeutics cannot be approached as “one size fits all” – as it is clear that each disease requires a unique approach to DNA editing. Guided by cutting-edge science and driven by a passion to deliver for patients, we have amassed one of the most talented and dedicated teams in the field to continue building upon the groundbreaking work of our founders.

The result is what we believe to be the most extensive toolbox of genomic editors in the industry to date, which has the PAM diversity to target more than 93% of the human genome—allowing us to begin with the underlying disease pathology and then choose an optimal editing approach for that disease. We believe this approach can yield important medicines and put cures within reach for patients with a wide range of genomic diseases.

Mission / Vision

Our Story

May: Closed on $15.6M Series A raise

2017

  • June: Optimized first therapeutic candidate for mammalian activity
  • July: Eight unique DNA-targeting CRISPR systems
  • August: Discovery of type V CRISPR-associated transposase system (Nature Reviews Microbiology)

2019

  • August: Expanded partnership with Vertex to include ex vivo engineered cell therapies
  • November: Closed $215 Million oversubscribed Series B Financing
  • December: Entered into gene editing technology licensing deal with Lonza

2021

  • January: Expanded strategic partnership with Vertex to include RT editing
  • October: Selected lead clinical development candidate ABO-101
  • October: Received feedback from European regulators paving the way to CTA filing
  • October: Established partnership with Ginkgo Bioworks to accelerate RT editor screening/optimization
  • December: Entered into a co-development/co-profit share agreement to secure access to novel AAV delivery vectors for CNS

2023

2016

Arbor founded by Feng Zhang, David Walt, David Scott, and Winston Yan

2018

  • March: Cas13d is a compact RNA-targeting CRISPR nuclease (Mol Cell)
  • December: Executed Vertex research collaboration and license agreement
  • December: Discovered additional subtypes of type V CRISPR-Cas systems (Science)

2020

  • March: Multiple nuclease candidates demonstrate mammalian activity
  • September: Engineering efforts yield candidates with Cas9 equivalent activity

2022

  • February: Entered license agreement with Edigene to advance ex vivo engineered cell therapy programs
  • May: Engineered Cas12i2 as platform for genome editing (Nature Communications)
  • August: Established partnership with Acuitas Therapeutics for LNPs for rare liver diseases
  • September: Named one of Fierce Biotech’s “Fierce 15”

2016

Arbor founded by Feng Zhang, David Walt, David Scott, and Winston Yan

2017

May: Closed on $15.6M Series A raise

2018

  • March: Cas13d is a compact RNA-targeting CRISPR nuclease (Mol Cell)
  • December: Executed Vertex research collaboration and license agreement
  • December: Discovered additional subtypes of type V CRISPR-Cas systems (Science)

2019

  • June: Optimized first therapeutic candidate for mammalian activity
  • July: Eight unique DNA-targeting CRISPR systems
  • August: Discovery of type V CRISPR-associated transposase system (Nature Reviews Microbiology)

2020

  • March: Multiple nuclease candidates demonstrate mammalian activity
  • September: Engineering efforts yield candidates with Cas9 equivalent activity

2021

  • August: Expanded partnership with Vertex to include ex vivo engineered cell therapies
  • November: Closed $215 Million oversubscribed Series B Financing
  • December: Entered into gene editing technology licensing deal with Lonza

2022

  • February: Entered license agreement with Edigene to advance ex vivo engineered cell therapy programs
  • May: Engineered Cas12i2 as platform for genome editing (Nature Communications)
  • August: Established partnership with Acuitas Therapeutics for LNPs for rare liver diseases
  • September: Named one of Fierce Biotech’s “Fierce 15”

2023

  • January: Expanded strategic partnership with Vertex to include RT editing
  • October: Selected lead clinical development candidate ABO-101
  • October: Received feedback from European regulators paving the way to CTA filing
  • October: Established partnership with Ginkgo Bioworks to accelerate RT editor screening/optimization
  • December: Entered into a co-development/co-profit share agreement to secure access to novel AAV delivery vectors for CNS

Founders

Board of Directors

Scientific Advisory Board

  • Clotilde Lagier-Tourenne, M.D., Ph.D.

    Associate Professor of Neurology, Harvard Medical School; Associate in Neuroscience, Massachusetts General Hospital; Healey Family ALS Endowed Chair for Research

  • Le Cong, Ph.D.

    Assistant Professor of Pathology and Genetics, Stanford University

  • Britt Adamson, Ph.D.

    Assistant Professor of Molecular Biology and the Lewis-Sigler Institute for Integrative Genomics, Princeton University

  • Guangping Gao, Ph.D.

    Co-Director, Li Weibo Institute for Rare Disease Research; Director, Horae Gene Therapy Center and Viral Vector Core; Professor of Microbiology and Physiological Systems; Penelope Booth Rockwell Professor in Biomedical Research, University of Massachusetts Medical School

  • Malin Parmer, Ph.D.

    Professor, New York Stem Cell Foundation - Robertson Investigator; Research Team Manager, Professor, Developmental Regenerative Neurobiology, Lund University

  • Kevin Alexander, M.D., F.A.C.C., F.H.F.S.A.

    Assistant Professor of Medicine - Cardiovascular Medicine, Stanford University School of Medicine

  • Patrick Hsu, Ph.D.

    Assistant Professor and Deb Faculty Fellow, University of California, Berkeley

  • Yizhou Dong, Ph.D.

    Associate Professor Pharmaceutics and Pharmacology, Ohio State University, College of Pharmacy

  • Stephen Goff, Ph.D.

    Higgins Professor of Microbiology & Immunology and Biochemistry & Molecular Biophysics, Columbia University

  • Osamu Nureki, Ph.D.

    Professor, Dept of Biophysics and Biochemistry, Dept of Biological Sciences, Graduate School of Science, University of Tokyo School of Science

Investors

We have attracted capital from top investors in the industry including:

  • Janus Henderson Investors