Arbor Biotechnologies

Company Overview

Arbor Biotechnologies focuses on next-generation gene editing to treat genetic diseases. The company is at the forefront of developing a diverse toolbox of novel gene editors aimed at advancing genetic medicine. Arbor Biotechnologies aims to create genetic medicines that transition from treatment to functional cures and prevention. Their mission is to develop these therapies through the discovery of programmable DNA editors.

Technologies and Platforms

Arbor Biotechnologies employs cutting-edge technologies, including lipid nanoparticles (LNPs) for liver-targeted gene editing and adeno-associated virus (AAV) vectors for CNS-targeted gene editing. Their proprietary discovery engine leverages AI/ML optimization to advance novel editing approaches. They have discovered 9 proprietary gene editors and identified six times more nuclease families than those reported in the literature. Additionally, the company has developed Cas12i2 as a platform for genome editing.

Research and Development Pipeline

Arbor Biotechnologies has a robust pipeline targeting liver and central nervous system (CNS) diseases. They have chosen primary hyperoxaluria (PH) and transthyretin amyloidosis (ATTR) as their initial clinical indications. The company's wholly owned portfolio includes in vivo targeted genetic medicines and partnerships for ex vivo applications. Arbor Biotechnologies envisions a world free of disease through genetic medicines that shift from treatment to functional cures and prevention.

Strategic Partnerships

Arbor Biotechnologies has established several strategic partnerships to advance its technologies and expand its impact. These include a co-development/co-profit share agreement for novel AAV delivery vectors for CNS, a partnership with Ginkgo Bioworks to accelerate RT editor screening and optimization, and a gene editing technology licensing deal with Lonza. They have also expanded their partnership with Vertex to include ex vivo engineered cell therapies and RT editing, and entered a license agreement with Edigene to advance ex vivo engineered cell therapy programs.

Recognition and Awards

In 2022, Arbor Biotechnologies was named one of Fierce Biotech’s “Fierce 15,” recognizing its leadership and innovation in the biotech sector. This accolade highlights their commitment to developing groundbreaking gene editing therapies and solidifies their reputation in the industry as a leading biotech company.

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