Drug intelligence / Profile preview

T- and B-cell depleted haploidentical hematopoietic stem cells (St. Jude Children's Research Hospital)

Development stage
Unknown
Lead developer
St. Jude Children's Research Hospital
Modality
Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

T- and B-cell depleted haploidentical hematopoietic stem cells (St. Jude Children's Research Hospital) is a cell therapy intervention developed for the treatment of Wiskott-Aldrich syndrome (WAS), a rare X-linked primary immunodeficiency. The procedure involves the transplantation of hematopoietic stem cells from a partially HLA-matched (haploidentical) family donor. To mitigate the risks of graft-versus-host disease (GVHD) and Epstein-Barr virus-associated post-transplant lymphoproliferative disorder (PTLPD), the donor graft is processed using the CliniMACS selection system to deplete T-lymphocytes (CD3+) and B-lymphocytes (CD19+). A controlled number of T-cells is subsequently added back to the graft prior to infusion to facilitate immune reconstitution and prevent graft failure. This approach aims to restore functional Wiskott-Aldrich syndrome protein (WASP) expression and normal hematopoietic function in patients who lack a fully matched related or unrelated donor.

Other names
T- and B-cell depleted haploidentical HSCTHaploidentical HSCT for Wiskott-Aldrich SyndromeT- and B-cell depleted haploidentical hematopoietic stem cells-St. Jude Children's Research Hospital-Wiskott-Aldrich syndrome

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