Drug intelligence / Profile preview

Haploidentical Hematopoietic Cell Transplantation-Johns Hopkins All Children's Hospital-primary immunodeficiency disease-inherited metabolic disorder-histiocytic disorder

Development stage
Unknown
Lead developer
Johns Hopkins All Children’s Hospital
Modality
Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

This investigational cell therapy procedure involves the transplantation of hematopoietic stem cells from a haploidentical (half-matched) donor, which have been processed ex vivo to selectively deplete TCR alpha/beta (TCRαβ+) T-cells and CD19+ B-cells. Developed and studied by Johns Hopkins All Children's Hospital, the procedure utilizes the CliniMACS system to remove the primary mediators of graft-versus-host disease (GVHD) while retaining CD34+ stem cells, natural killer (NK) cells, and TCR gamma-delta (TCRγδ+) T-cells. This approach aims to provide rapid hematopoietic recovery and immune reconstitution with a reduced risk of GVHD in pediatric patients suffering from primary immunodeficiency diseases, inherited metabolic disorders, and histiocytic disorders.

Other names
TCR alpha beta T-cell and CD19 B-cell depleted haploidentical transplantationHaploidentical Hematopoietic Cell TransplantationTCR alpha beta/CD19 depleted haploidentical HCT
02

Targets

CD19 (B lymphocyte antigen CD19)TCR (T cell antigen receptor complex)

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