Drug intelligence / Profile preview

Dualase

Development stage
Preclinical
Lead developer
Specific Biologics
Modality
Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Inhalation, Intrathecal, Intravenous, Ophthalmic
01

Overview

Dualase is a proprietary gene editing platform developed by Specific Biologics. It utilizes a novel double-cutter nuclease design that enables precise and efficient DNA editing by creating two targeted cuts in the genome. This approach allows for both precise DNA deletions (to disrupt genes) and increased repair (to correct genes), leveraging the cell’s natural DNA repair pathways. The technology aims to address genetic mutations that are not easily targeted with existing gene editors, offering potential advantages in specificity and therapeutic outcomes. Preclinical development is focused on diseases such as cystic fibrosis, as well as conditions affecting the liver, eyes, and central nervous system[2][6][8][10].

Brand names
Dualase
Other names
Dualase gene editing programDualase genome editorDualase platform
02

Targets

gDNA (Genomic DNA at disease-causing loci)

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