The ENCEL system: a somatic cell protein delivery system.

A wide variety of somatic cells are being explored for the introduction of foreign genes with a view toward gene therapy. A prime requirement for successful gene therapy is the sustained expression, effective dosing, and systemic delivery of the therapeutic gene product. Microvascular endothelial cells offer several advantages over other cell types as a somatic cell gene delivery vehicle in that they provide direct secretion of protein into the blood stream and they are amendable to highly stable retroviral-based protein expression. Importantly, they also offer a large surface volume to size ratio in that they can be induced with angiogenic factors to form organized capillary-like structures in vitro when grown in a three dimensional culture system using collagen gels. These genetically-modified capillary endothelial cells (the ENCEL system) maintained in collagen gels can be stably transplanted and removed. The unique biological properties of microvascular capillary endothelial cells allows the ENCEL system to provide large numbers of cells in a small volume which offers the highly desired opportunity for providing a sustained and effective dose of a therapeutic protein. Alexion is currently applying its Unigraft immunotherapeutic and engineering technologies to commercialize a non-human ENCEL system acceptable for implantation into any patient.