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CCR5-Uco-hetTALEN
- Development stage
- Preclinical
- Lead developer
- AGCT Bio
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
CCR5-Uco-hetTALEN is an optimized transcription activator-like effector nuclease (TALEN) designed to target and disrupt the C-C chemokine receptor type 5 (CCR5) gene, which encodes the primary co-receptor utilized by CCR5-tropic HIV-1 to enter host immune cells. Developed by the University Medical Center Hamburg-Eppendorf and licensed to AGCT Bio, this gene-editing tool is delivered as mRNA via electroporation. It features an obligatory heterodimeric FokI cleavage domain that significantly reduces off-target activity (such as at homodimeric sites) while maintaining high-efficiency on-target knockout of CCR5 in CD4+ T cells and hematopoietic stem cells (HSCs). This approach aims to generate HIV-resistant immune cells as a functional cure for HIV-1 infection.
02
Targets
CCR5 (C-C chemokine receptor type 5)
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