allogeneic hematopoietic stem cells (Columbia University, c03422)

This investigational cell therapy involves the transplantation of HLA-matched allogeneic hematopoietic stem cells (HSCs) or progenitor cells (HPCs) for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). RDEB is a severe inherited blistering disease caused by mutations in the *COL7A1* gene, which results in a deficiency of functional type VII collagen at the dermal-epidermal junction. The therapeutic approach utilizes a reduced-intensity conditioning (RIC) regimen—typically involving agents like busulfan, fludarabine, and alemtuzumab—to facilitate donor cell engraftment. Following transplantation, donor-derived hematopoietic cells are intended to repopulate the patient's system and potentially migrate to the skin, where they can restore the expression of type VII collagen, thereby improving skin integrity, reducing blistering, and enhancing wound healing.

Other names

allogeneic hematopoietic progenitor cellsAlloSCTallogeneic bone marrow transplantation