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CRISPR-based gene therapy using Incisive Delivery System
- Development stage
- Preclinical
- Lead developer
- Incisive Genetics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous, Intrathecal
01
Overview
The CRISPR-based gene therapy using the Incisive Delivery System is a therapeutic approach that employs a proprietary lipid nanoparticle (LNP) platform to deliver pre-assembled active CRISPR ribonucleoprotein (RNP) complexes—comprising Cas9 protein and guide RNA—directly into target cells. This system enables rapid on-target genome editing with transient presence of the editing machinery, which is naturally degraded and cleared by cellular mechanisms. The technology is designed for in vivo applications and aims to improve the safety and efficacy of genetic therapies by minimizing off-target effects and immune responses associated with viral vectors. Developed primarily for therapeutic gene editing, this platform can be adapted for various genetic diseases depending on the guide RNA used[1][2][6].
- Brand names
- Incisive Delivery System
- Other names
- IDSIncisive Genetics CRISPR delivery platformlipid nanoparticle-based CRISPR delivery system
02
Targets
Protospacer (Genomic DNA target sequence)
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