AMB-301

**AMB-301** is a first-in-class **AAV vector-based gene therapy** encoding **E-selectin**, a cell adhesion molecule, developed by **Ambulero**, a spinout from the University of Miami Miller School of Medicine. It promotes **therapeutic angiogenesis**, tissue regeneration, and improved limb function in damaged vascular tissues, based on over a decade of preclinical research by Drs. Omaida C. Velazquez and Zhao-Jun Liu. The therapy targets **Buerger’s Disease** (thromboangiitis obliterans), a rare vasculitis causing severe limb ischemia and amputation risk in young patients, with no effective current treatments; it has received **FDA Orphan Drug Designation** and positive Pre-IND feedback, with IND-enabling studies ongoing as of June 2025 and an IND filing planned by late 2024.[1][2][3][5]