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CCR5-specific zinc finger nucleases
- Development stage
- Unknown
- Lead developer
- Sangamo Therapeutics
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Intravenous
01
Overview
CCR5-specific zinc finger nucleases (ZFNs) are an ex vivo gene editing therapy designed to treat HIV/AIDS by disrupting the CCR5 gene. CCR5 is a critical co-receptor used by most strains of HIV-1 to enter and infect CD4+ T cells. By using ZFNs—engineered proteins consisting of a DNA-binding zinc finger domain fused to a FokI endonuclease—to create targeted double-strand breaks in the CCR5 locus, the therapy induces mutations that prevent the expression of functional CCR5 receptors. This creates a population of HIV-resistant immune cells, mimicking the natural resistance found in individuals with the CCR5Δ32 mutation. Developed primarily by Sangamo Therapeutics, the most prominent candidate is SB-728, which has been evaluated in clinical trials using both autologous CD4+ T cells (SB-728-T) and hematopoietic stem/progenitor cells (SB-728-HSPC) delivered via viral vectors or mRNA.
- Other names
- CCR5-ZFNCCR-5-ZFNCCR 5-ZFNzinc finger nucleases targeting CCR5
02
Targets
CCR5 (C-C chemokine receptor type 5)
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