Company intelligence / Profile preview
Arbor Biotechnologies
Cambridge, Massachusetts, USAarbor.bio ↗
- Ownership
- Private
- Development stage
- Other
01
Overview
ABO‑101 demonstrated promising in vivo non-human primate data supporting its advancement into clinical trials for PH1. The company’s pipeline includes additional preclinical programs targeting ALS via STMN2 disruption and cardiometabolic disease via PCSK9 silencing using proprietary compact nucleases.
- Therapeutic areas
- Rare DiseasesNeurologyCardiovascular
- Modalities
- In vivo gene editing (CRISPR-based)Programmable DNA editors (including novel nucleases such as Cas12i2 and ABR series)Ex vivo cell therapy platforms
- Industry
- Biotech
02
Drug pipeline
9 assets8 more assets in the full pipeline.
03
Partnerships
Vertex Pharmaceuticals – research collaboration/licensing for ex vivo engineered cell therapiesLonza – licensing deal for technologyEdigene – license agreement to advance ex vivo engineered cell therapy programsPartnership with 4D Molecular Therapeutics (“4DMT”) to co-develop/commercialize CNS genetic medicines
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