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Moogene Medi LNP-CRISPR RNP therapy
- Development stage
- Preclinical
- Lead developer
- Moogene Medi
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous, Intramuscular, Topical, Transdermal
01
Overview
Moogene Medi LNP-CRISPR RNP therapy is an experimental gene-editing therapeutic being developed by Moogene Medi for the treatment of colorectal and pancreatic cancers, particularly those exhibiting resistance to standard therapies due to specific genetic mutations. The platform utilizes lipid nanoparticles (LNPs) to deliver CRISPR-Cas9 and single-guide RNA (sgRNA) as pre-assembled ribonucleoprotein (RNP) complexes. This RNP-based delivery method is designed to facilitate immediate gene editing upon entry into the target cell and minimize the duration of Cas9 expression, thereby potentially reducing off-target effects compared to DNA or mRNA-based delivery. The therapy is currently in the preclinical stage of development and aims to overcome anticancer drug resistance by directly editing pathogenic mutations.
- Other names
- LNP for gene editing therapyCRISPR-Cas9/sgRNA RNP-LNP
02
Targets
SRD5A2 (Steroid 5-alpha-reductase type II)
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