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Company intelligence / Profile preview
Arbor
Cambridge, Massachusetts, United Statesarbor.bio ↗
- Ownership
- Private
- Development stage
- Other
01
Overview
Arbor Biotechnologies has demonstrated preclinical efficacy and pharmacology for its lead gene editing therapy ABO-101. ABO-101 targets primary hyperoxaluria type 1 and is supported by non-human primate data and regulatory approvals for first-in-human studies in the UK and EU. The CNS program (ALS) has shown preclinical proof-of-concept
- Therapeutic areas
- NeurologyRare Diseases
- Modalities
- Gene therapyCRISPR-based therapyProgrammable DNA editorRNA therapy
- Industry
- Biotech
02
Partnerships
Vertex Pharmaceuticals (expanded for gene editing and ex vivo cell therapies)Lonza (gene editing technology licensing)Ginkgo Bioworks (R&D for RT editor screening/optimization)Allogene Therapeutics (CAR-T editing for autoimmune disease)4D Molecular Therapeutics (AAV-delivered CRISPR/Cas therapies for CNS, co-development/co-commercialization)Edigene (license agreement for ex vivo engineered cell therapy)
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