Arbor Biotechnologies

Arbor Biotechnologies is dedicated to advancing next-generation gene editing technologies to treat genetic diseases. The company has developed a diverse toolbox of novel gene editors and employs lipid nanoparticles (LNPs) for liver-targeted gene editing and adeno-associated virus (AAV) for CNS-targeted gene editing. Arbor’s proprietary discovery engine, enhanced by AI/ML optimization, has led to the discovery of nine proprietary gene editors and six times more nuclease families than what is published in the literature. The company maintains a wholly owned portfolio of editing technologies for in vivo targeted genetic medicines and partners with leading companies for ex vivo applications. Arbor’s initial clinical indications include primary hyperoxaluria (PH) and transthyretin amyloidosis (ATTR). Recognized as one of Fierce Biotech’s “Fierce 15” in 2022, Arbor has entered into several key partnerships and agreements, including a co-development/co-profit share agreement for novel AAV delivery vectors for CNS, a partnership with Ginkgo Bioworks to accelerate RT editor screening/optimization, a gene editing technology licensing deal with Lonza, and an expanded partnership with Vertex for ex vivo engineered cell therapies and RT editing. Additionally, Arbor has a license agreement with Edigene to advance ex vivo engineered cell therapy programs. The company has developed Cas12i2 as a platform for genome editing and is driven by a mission to develop genetic medicines through the discovery of programmable DNA editors, envisioning a world free of disease by shifting from treatment to functional cure and prevention.