Arbor Biotechnologies
Arbor Biotechnologies is a next-generation gene editing company dedicated to discovering and developing potentially curative genomic medicines. With an extensive toolbox of proprietary gene editors, the company focuses on advancing therapies for genetic diseases, especially in liver and CNS areas, through innovative technologies like CRISPR and AI-driven discovery. Arbor aims to treat a broad spectrum of patients, from ultra-rare to common genetic conditions, by leveraging its unique suite of gene editing tools and strategic partnerships.
Industries
Nr. of Employees
medium (51-250)
Patents
Gene editing systems comprising an RNA guide targeting lactate dehydrogenase a (LDHA) and uses thereof
US-11939607-B2
View Details
Gene editing systems comprising an RNA guide targeting lactate dehydrogenase a (LDHA) and uses thereof
US-11939607-B2
View DetailsProducts
ABO-101
Investigational, one-time liver-directed gene editing therapeutic designed to permanently reduce HAO1 expression in hepatocytes to lower oxalate production in primary hyperoxaluria type 1.
ABO-101
Investigational, one-time liver-directed gene editing therapeutic designed to permanently reduce HAO1 expression in hepatocytes to lower oxalate production in primary hyperoxaluria type 1.
Services
Partnership-based R&D to optimize gene editors using automated foundry workflows, high-throughput mammalian screening, and iterative AI-guided protein engineering.
Granting third parties rights to use proprietary gene editing technologies in bioproduction products and cell therapy programs under license agreements.
Joint development and co-commercialization arrangements to combine compact editor payloads with customized AAV vectors for CNS-targeted therapeutics.
Partnership-based R&D to optimize gene editors using automated foundry workflows, high-throughput mammalian screening, and iterative AI-guided protein engineering.
Granting third parties rights to use proprietary gene editing technologies in bioproduction products and cell therapy programs under license agreements.
Joint development and co-commercialization arrangements to combine compact editor payloads with customized AAV vectors for CNS-targeted therapeutics.
Expertise Areas
- Next-generation gene editing platform development
- Protein engineering and directed evolution
- AI/ML-driven biological discovery
- High-throughput mammalian screening
Key Technologies
- CRISPR-Cas nuclease discovery (including Type V and RNA-targeting systems)
- Reverse transcriptase (RT)-based editing
- CRISPR transposase approaches
- AAV vector delivery
News & Updates
Arbor announced that the FDA has cleared the IND for ABO-101, a gene editing therapeutic for primary hyperoxaluria type 1, with a planned clinical trial to evaluate safety, tolerability, and efficacy.
Arbor announced the discovery of new CRISPR-Cas systems, including Cas12g, Cas12h, and Cas12i, published in Science, and the appointment of Paul Meister to its Board of Directors.
Published in Science, Arbor described its systematic approach for identifying, screening, and characterizing novel proteins, including the smallest known RNA-targeting CRISPR enzyme, Cas12g.
Arbor announced that the FDA has cleared the IND for ABO-101, a gene editing therapeutic for primary hyperoxaluria type 1, with a planned clinical trial to evaluate safety, tolerability, and efficacy.
Arbor announced the discovery of new CRISPR-Cas systems, including Cas12g, Cas12h, and Cas12i, published in Science, and the appointment of Paul Meister to its Board of Directors.
Published in Science, Arbor described its systematic approach for identifying, screening, and characterizing novel proteins, including the smallest known RNA-targeting CRISPR enzyme, Cas12g.