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R-66S gRNA
- Development stage
- Unknown
- Lead developer
- Excision BioTherapeutics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
R-66S gRNA is a specific guide RNA component developed by Excision BioTherapeutics for use in CRISPR-based gene editing therapies targeting HIV-1. It is designed to recognize and bind to a highly conserved sequence within the Long Terminal Repeat (LTR) region of the HIV-1 proviral DNA. In the therapeutic candidate EBT-101, R-66S is one of two guide RNAs (the other being S-1) that direct the Staphylococcus aureus Cas9 (SaCas9) nuclease to excise a large segment of the HIV-1 genome, effectively inactivating the virus and preventing its replication. This dual-gRNA approach is intended to minimize the risk of viral escape and ensure comprehensive removal of the provirus from latent reservoirs. EBT-101, which incorporates R-66S, is delivered via an adeno-associated virus serotype 9 (AAV9) vector and is currently being evaluated in clinical trials as a potential functional cure for individuals living with HIV-1.
- Other names
- HIV-1 LTR-targeting guide RNAHIV1 LTR-targeting guide RNAHIV 1 LTR-targeting guide RNA
02
Targets
Human immunodeficiency virus type 1 long terminal repeat
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