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RNAfix
- Development stage
- Preclinical
- Lead developer
- Shape Therapeutics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
RNAfix is a proprietary programmable RNA editing platform developed by Shape Therapeutics. It utilizes DNA-encoded guide RNAs (gRNAs) to recruit the endogenous enzyme adenosine deaminase acting on RNA (ADAR), enabling precise and efficient A-to-I (adenosine-to-inosine) base editing at specific sites in target RNAs. This approach allows for correction of disease-causing mutations or modulation of gene expression without altering genomic DNA, offering a safer alternative to CRISPR-based genome editing. The system achieves high efficiency (>95% in mouse brain, >90% in non-human primate brain), high specificity with minimal off-target effects, and durable effects lasting at least six months post-dose. Delivery is achieved via adeno-associated virus (AAV) vectors capable of crossing the blood-brain barrier, making it particularly suitable for central nervous system disorders such as Rett syndrome, Parkinson’s disease, and Alzheimer’s disease[1][2][4][7].
- Brand names
- RNAfix
- Other names
- RNAfix editing platformShapeTX RNAfix
02
Targets
ADARB1 (Adenosine deaminase, RNA-specific, B1)
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