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Drug intelligence / Profile preview
AAV-CBFβ
- Development stage
- Preclinical
- Lead developer
- Tulane University
- Modality
- Gene Therapies
- Administration
- Intraarticular, Subcutaneous
01
Overview
AAV-CBFβ is an adeno-associated virus (AAV)-based gene therapy designed to deliver and overexpress core-binding factor subunit beta (CBFβ). CBFβ is a non-DNA-binding transcription cofactor that heterodimerizes with Runt-related transcription factors (RUNX1, RUNX2, and RUNX3) to enhance their DNA-binding affinity and stability. Preclinical studies conducted by academic institutions, including Zhejiang University, Tulane University, and the University of Alabama at Birmingham, have demonstrated that AAV-CBFβ can promote osteogenesis, chondrocyte differentiation, and cartilage regeneration. It has shown therapeutic potential in animal models of osteoarthritis, osteoporosis, and age-related bone fracture repair by modulating signaling pathways such as Wnt/β-catenin, Hippo/YAP, and TGF-β.
- Other names
- AAV-CBFbetaAAV-CbfbAAV-Cbfβ
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