AAV2-Foxf1

AAV2-Foxf1 is an experimental gene therapy candidate being investigated for the treatment of pulmonary arterial hypertension (PAH). Developed by researchers at Stanford University, the therapy utilizes an adeno-associated virus serotype 2 (AAV2) vector—often engineered with a targeting peptide for the pulmonary arterial endothelium—to deliver the FOXF1 (Forkhead Box F1) gene. FOXF1 is a transcription factor critical for endothelial cell homeostasis and DNA repair. In PAH, reduced FOXF1 expression (often linked to BMPR2 mutations) leads to persistent DNA damage and impaired angiogenesis. Restoration of FOXF1 via AAV2-Foxf1 has been shown in preclinical models to attenuate DNA damage, restore expression of angiogenic genes such as CLDN5 and VEGFR2, and reverse pulmonary hypertension.