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AAV1-BMP3
- Development stage
- Preclinical
- Lead developer
- Icahn School of Medicine
- Modality
- Gene Therapies
- Administration
- Inhalation
01
Overview
AAV1-BMP3 is an adeno-associated virus serotype 1 (AAV1) vector-based gene therapy designed to overexpress Bone Morphogenetic Protein 3 (BMP3) in lung tissue. Developed by researchers at the Icahn School of Medicine at Mount Sinai and Virginia Tech, it is being investigated for the treatment of pulmonary arterial hypertension (PAH). BMP3 is a member of the transforming growth factor-beta (TGF-β) superfamily that is downregulated in PAH. By restoring BMP3 levels, the therapy aims to rebalance signaling pathways by activating the protective BMP/Smad1/5/8 axis and suppressing the pro-fibrotic TGF-β/Smad2/3 axis, thereby attenuating pulmonary vascular remodeling and right ventricular dysfunction.
02
Targets
α2,3-Sia (Sialylated cell-surface glycans bearing α2,3-linked sialic acid)ACVR2B (Activin receptor type IIB)ACVR2A (Activin receptor type 2A)
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