Ambulero’s gene and cell therapy programs are based on ground-breaking work from the laboratories of Dr. Omaida Velazquez, Chair and Professor of Surgery, the University of Miami Miller School of Medicine, and Dr. Zhao-Jun Liu, Professor of Surgery, University of Miami Miller School of Medicine. Dr. Velazquez is the principal investigator of NIH-funded research to understand vascular biology better including how new blood vessels grow in response to disease. This laboratory work has been selected for prestigious funding under NIH’s Vascular Interventions/Innovations and Therapeutic Advances (VITA) program.

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Ambulero is engineering a subpopulation of bone marrow stromal cells (mesenchymal cells or “MSCs”) to express E-selectin: a cell surface adhesion molecule typically expressed by endothelial cells lining blood vessels. When injected into animal models of limb tissue damage (ischemia), the engineered cells help heal damaged limbs significantly better than sham controls. We call the engineered cells “supercharged MSCs” to denote the positive effect they have on healing and restoration of running ability in these animals.

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Other forms of vascular damage involve severe inflammation and may be more amenable to gene therapy approaches in which supercharged cells are not used. In this approach, E-selectin is administered to areas of tissue damage without use of supercharged MSCs. We are developing a gene therapy approach using clinically established AAV vectors to deliver E-selectin to where it is needed. Ongoing non-clinical work with our therapeutic lead product (AMB-201) is promising and our goal is to use this approach to treat ischemic indications with large patient numbers such as chronic limb ischemia (CLI).

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For more background information on our therapy approaches, please refer to scientific publications from the laboratories of Dr. Omaida Velazquez and Dr. Zhao-Jun Liu.

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