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PBGENE-CNS
- Development stage
- Preclinical
- Lead developer
- Precision BioSciences
- Modality
- Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous, Intrathecal
01
Overview
PBGENE-CNS is an investigational gene editing therapy developed by Precision BioSciences. It is in preclinical development and targets diseases of the nervous system. The drug utilizes the ARCUS gene editing platform, which is based on a proprietary engineered meganuclease designed for precise genome modification. While specific mechanisms or disease targets for PBGENE-CNS have not been disclosed, it follows the same technological approach as other PBGENE programs, which use mRNA encoding ARCUS nucleases delivered via lipid nanoparticles to achieve targeted in vivo gene editing[2]. The primary indication area is central nervous system (CNS) disorders.
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