Drug intelligence / Profile preview

gene-modified HIV-protected hematopoietic stem cells

Development stage
Unknown
Lead developer
Fred Hutchinson Cancer Center
Modality
Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

Gene-modified HIV-protected hematopoietic stem cells are autologous hematopoietic stem cells (HSCs) that have been genetically engineered to resist HIV infection. This therapy involves collecting CD34+ hematopoietic stem/progenitor cells from an HIV-positive individual, genetically modifying them with anti-HIV genes, and then reinfusing them back into the patient. The modified stem cells can differentiate into immune cells that are resistant to HIV infection and may also be able to destroy HIV-infected cells. This approach aims to provide a once-only treatment that reduces viral load, preserves the immune system, and potentially avoids cumulative toxicities associated with highly active antiretroviral therapy (HAART).

Other names
OZ1OZ-1OZ 1anti-HIV gene-modified hematopoietic stem cells
02

Targets

CCR5 (C-C chemokine receptor type 5)

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